Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
COVID Protection After Transplant - Sanofi GSK (CPAT-SG) Study (CPAT-SG)
An open label, non-randomized pilot study in kidney transplant recipients who received a
completed primary series and bivalent booster of mRNA based COVID-19 vaccine and have =<2500
U/mL SARS-CoV-2 S antibody concentration using the Roche Elecsys(R) anti-RBD assay. Up to 80
participants will be enrolled in this study. Eligible participants will receive a dose of the
Sanofi-GSK monovalent (B.1.351) CoV2 preS dTM-AS03 COVID-19 vaccine candidate..
The primary objective is to determine whether a booster dose of the Sanofi-GSK monovalent
(B.1.351) CoV2 preS dTM-AS03 COVID-19 vaccine will elicit an increased SARS-CoV-2 antibody
response in participants who have failed to maintain an antibody titer >2500 U/mL (using the
Roche Elecsys(R) anti-RBD assay) to 2 or more doses of mRNA based COVID-19 vaccine
Jacqueline Garonzik Wang
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05518487
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Inclusion Criteria:
1. Able to understand and provide informed consent
2. Individual ≥ 18 years of age.
3. Recipient of kidney transplant >=12 months prior to enrollment, without treated
allograft rejection in the 6 months preceding enrollment
4. Maintenance immunosuppressive regimen consisting of CNI and mycophenolate mofetil or
mycophenolate, with or without <= 5mg/day prednisone or equivalent
5. Received completed primary series (3 doses) of mRNA vaccine (either the Moderna
COVID-19 vaccine or Pfizer-BioNTech COVID-19 vaccine) as specified in the respective
package inserts
6. Receipt a COVID-19 bivalent mRNA booster (Moderna or Pfizer-BioNTech) >30 days prior
to enrollment.
7. Serum antibody titer up to 2500 U/mL at >=30 days from the last dose of mRNA COVID-19
vaccine and =>30 days following receipt of a monoclonal antibody product or
convalescent plasma for COVID-19, measured using the Roche Elecsys(R) anti-SARS-CoV-2
S assay
8. Platelet count greater than 30,000/cu mm must be confirmed in participants with a
known history of bleeding disorder or thrombocytopenia (platelet count <50,000/cu mm)
9. A female participant is eligible to participate if she is not pregnant or
breastfeeding and one of the following conditions applies:
1. Is of non-childbearing potential. To be considered of non-childbearing potential,
a female must be post-menopausal for at least 1 year or surgically sterile
OR
2. Is of childbearing potential and agrees to use an effective contraceptive method
or abstinence for 12 weeks post vaccine and while taking mycophenolate
mofetil/mycophenolic acid
Exclusion Criteria:
1. Recipient of any number of doses of any COVID vaccine product other than the Moderna
COVID-19 vaccine or the Pfizer-BioNTech COVID-19 vaccine
2. Recipient of any organ other than a kidney
3. Known current or prior Donor Specific Antibody (DSA)
4. Any change in transplant immunosuppression regimen (drug or dose) in response to
suspected or proven rejection within the last 6 months
5. Known diagnosis of COVID-19 since last antibody test
6. Receipt of a monoclonal antibody product or convalescent plasma within the last 30
days
7. Known history of hypersensitivity to any of the vaccine components, or history of a
life-threatening reaction to a vaccine containing any of the same substances.
(components listed in Section 6, and the CoV2 and AS03 Investigator's Brochure)
8. Bleeding disorder, or receipt of anticoagulants in the past 21 days preceding
inclusion, contraindicating intramuscular (IM) vaccination based on Investigator's
judgment
9. Moderate or severe acute illness/infection (according to investigator judgment) on the
day of vaccination or febrile illness (temperature >=38.0°C [>=100.4°F]). A
prospective participant should not be included in the study until the condition has
resolved or the febrile event has subsided
10. Receipt of any vaccine in the 30 days preceding the study vaccine or planned vaccines
in the 30 days following the study vaccine
11. Estimated Glomerular Filtration Rate <30mL/min/1.73m^2
12. Receipt of any cellular depleting agent (e.g. Antithymocyte globulin (ATG), Rituximab,
Alemtuzumab, Cyclophosphamide) within 12 months preceding enrollment
13. Receiving systemic immunomodulatory medication(s) for any condition other than
transplant
14. Any uncontrolled active infection
15. Infection with human immunodeficiency virus (HIV)
16. Maintenance immunosuppressive regimen that includes anything other than a CNI,
mycophenolate/mycophenolate mofetil, and =< 5mg/day prednisone or equivalent
17. Recent (within one year) or ongoing treatment for malignancy, except for definitive
surgical treatment of localized skin cancers
18. Any unstable acute or chronic illness, treatments, or findings which, in the opinion
of the investigator, may pose additional risks from participation in the study, may
interfere with the c candidate's ability to comply with study requirements or may
impact the quality or interpretation of the data obtained from the study
COVID-19, Kidney Transplant, Kidney replaced by transplant, Other, Transplant
The study objective for the Phase 2 of this research is to demonstrate and confirm the
substantial time savings that can be obtained using cone beam computed tomography (CB-CT) for
both complete image acquisition and rapid image reconstruction in a Direct to Angio paradigm
(One Stop Shop) for selected acute ischemic stroke (AIS) patients.
Beverly Aagaard
All
18 Years to 99 Years old
N/A
This study is NOT accepting healthy volunteers
NCT05536895
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Inclusion Criteria:
1. Patients with acute ischemic stroke presenting within 24 hours of onset
2. Patients that present with a large artery occlusion
3. Adults 18 years of age or older.
4. Patients of childbearing potential must not be pregnant.
5. National Institutes of Health Stroke Scale (NIHSS) of <8
6. No severe co-morbidities
Exclusion Criteria:
1. Patients that are pregnant
2. History of severe renal disease (e.g. stage 4-5)
3. History of renal transplant
Oral N-acetylcysteine for Retinitis Pigmentosa (NAC Attack)
Retinitis pigmentosa (RP) is an inherited retinal degeneration caused by one of several
mistakes in the genetic code. Such mistakes are called mutations. The mutations cause
degeneration of rod photoreceptors which are responsible for vision in dim illumination
resulting in night blindness. After rod photoreceptors are eliminated, gradual degeneration
of cone photoreceptors occurs resulting in gradual constriction of side vision that
eventually causes tunnel vision. Oxidative stress contributes to cone degeneration.
N-acetylcysteine (NAC) reduces oxidative stress and in animal models of RP it slowed cone
degeneration. In a phase I clinical trial in patients with RP, NAC taken by month for 6
months caused some small improvements in two different vision tests suggesting that long-term
administration of NAC might slow cone degeneration in RP. NAC Attack is a clinical trial
being conducted at many institutions in the US, Canada, Mexico, and Europe designed to
determine if taking NAC for several years provides benefit in patients with RP.
Kimberly Stepien, MD
All
18 Years to 65 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05537220
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Inclusion Criteria:
General
• Ability and willingness to provide informed consent
• Age ≥ 18 and ≤65 years at time of signing Informed Consent Form
• Ability and willingness to comply with the study protocol and to participate in all
study visits and assessments in the investigator's judgement
• For candidates of childbearing potential: willingness to use a method of contraception
• Agreement not to take supplements other than vitamin A
Ocular Inclusion Criteria
• Both eyes must exhibit the RP phenotype with evidence of loss of night vision, gradual
constriction of visual fields, and maintenance of visual acuity;
• In addition, an eye must meet the following criteria to be included in the study:
• Gradable EZ on a horizontal SD-OCT scan through the fovea center with width ≤ 8000 µm
and ≥1500 µm and with well-defined truncation at both the nasal and temporal sides;
• BCVA ≥ ETDRS letter score of 61 (20/60 Snellen equivalent);
• Sufficiently clear ocular media and adequate pupillary dilation to allow good quality
images sufficient for analysis and grading by central reading center.
Exclusion Criteria:
General Exclusion Criteria
• Active cancer within the past 12 months, except for appropriately treated carcinoma in
situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with Gleason score
≤ 6 and stable prostate specific antigen for > 12 months
• Renal failure requiring renal transplant, hemodialysis, peritoneal dialysis, or
anticipated to require hemodialysis or peritoneal dialysis during the study
• Liver disease, cystic fibrosis, asthma, or chronic obstructive pulmonary disease
(COPD), history of thrombocytopenia not due to a reversible cause or other blood
dyscrasia
• Uncontrolled blood pressure (defined as systolic > 180 and/or diastolic > 100 mmHg
while at rest) at screening. If a patient's initial measurement exceeds these values,
a second reading may be taken 30 or more minutes later. If the patient's blood
pressure must be controlled by antihypertensive medication, the patient may become
eligible if medication is taken continuously for at least 30 days.
• History of other disease, physical examination finding, or clinical laboratory finding
giving reasonable suspicion that oral NAC may be contraindicated or that follow up may
be jeopardized
• Cerebrovascular accident or myocardial infarction within 6 months of screening
• Participation in an investigational study that involves treatment with any drug or
device within 6 months of screening
• Three relatives already enrolled in study
• Pregnant, breast feeding, or intending to become pregnant during the study treatment
period. Women of childbearing potential who have not had tubal ligation must have a
urine pregnancy test at screening.
• Known history of allergy to NAC
• Having taken NAC in any form in the past 4 months
• Phenylketonuria
• Fructose intolerance
• Glucose-galactose malabsorption
• Sucrase-isomaltase insufficiency
• Abnormal laboratory value including the value of alanine aminotransferase (ALT),
aspartate aminotransferase (AST), or bilirubin being greater than 1.5 x the upper
limit of normal
• Any major abnormal findings on blood chemistry, hematology, and renal function lab
tests that in the opinion of the Site Investigator and/or the Study Chair makes the
candidate not suitable to participate in the trial
• HIV or hepatitis B infection
Ocular Exclusion Criteria
• Evidence of cone-rod dystrophy or pattern dystrophy including focal areas of atrophy
or pigmentary changes in the central macula
• Cystoid spaces involving the fovea substantially reducing vision
• Glaucoma or other optic nerve disease causing visual field loss or reduced visual
acuity
• Intra ocular pressure >27 mm Hg from two measurements. If a patient's initial
measurement exceeds 27 mm Hg, a second reading must be taken.
• Any retinal disease other than RP causing reduction in visual field or visual acuity
• Any prior macular laser photocoagulation
• Intraocular surgery within 3 months prior to screening
• High myopia with spherical equivalent refractive error > 8 diopters. If an eye has had
cataract surgery or refractive surgery, a pre-operative refractive error spherical
equivalent > 8 diopters is an exclusion
• Any concurrent ocular condition that might affect interpretation of results
• History of uveitis in either eye
First-in-Human Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, as a Single Agent in Advanced Solid Tumor Patients and in Combination With Fulvestrant in Patients With Advanced Breast Cancer
This is an open-label, FIH study designed to evaluate the maximum tolerated dose, recommended
Phase 2 dose, safety, tolerability, PK, pharmacodynamics, and preliminary antineoplastic
activity of RLY-2608, in advanced solid tumor patients with a
Phosphatidylinositol-4,5-bisphosphate-3 kinase, catalytic subunit alpha (PIK3CA) mutation in
blood and/or tumor per local assessment. The study will evaluate RLY-2608 as a single agent
for patients with unresectable or metastatic solid tumors, RLY-2608 + fulvestrant and
RLY-2608 + fulvestrant + CDK4/6 inhibitor (palbociclib or ribociclib) combination arms for
patients with HR+ HER2- locally advanced or metastatic breast cancer. The RLY-2608 single
agent arm, RLY-2608 + fulvestrant combination arm, and triple combination arms will have 2
parts: a dose escalation (Part 1) and a dose expansion (Part 2).
Kari Wisinski, MD
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT05216432
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Key Inclusion Criteria
Patient has ECOG performance status of 0-1
One or more documented primary oncogenic PIK3CA mutation(s) in blood and/or tumor per local
assessment
•Other potentially oncogenic PIK3CA mutations may be considered but must be approved by
the Sponsor prior to enrollment.
Part 1 •Ability to provide archived tumor tissue or be willing to undergo pretreatment
tumor biopsy to assess PIK3CA status retrospectively Part 2 •Submit tumor tissue prior to
study drug initiation for determination of PIK3CA mutation retrospectively.
Key Inclusion for RLY-2608 Single Agent Arm
• [For Part 1]: Evaluable disease per RECIST v1.1
• [For Part 2]: Measurable disease per RECIST v1.1
• Disease that is refractory to standard therapy, intolerant to standard therapy, or has
declined standard therapy.
• Part 1- histologically or cytologically confirmed diagnosis of unresectable or
metastatic solid tumor
• Part 2 •Unresectable or metastatic solid tumor with PIK3CA mutation(s) and one of the
following tumor types:
Group 1: clear cell ovarian cancer Group 2: head and neck squamous cell carcinoma Group 3:
cervical cancer Group 4: other solid tumors, excluding colorectal, clear cell ovarian, head
and neck squamous cell, and cervical cancers Group 5: unresectable or metastatic solid
tumors with PIK3CA double mutations
Key Inclusion for Combination Arms
• [For Part 1 and Part 2]: Evaluable disease per RECIST v1.1
• Male or female with histologically or cytologically confirmed diagnosis of HR+, HER2-
unresectable or metastatic breast cancer that is not amenable to curative therapy.
Females may be postmenopausal, premenopausal, or perimenopausal. Premenopausal or
perimenopausal females must have a histologically or cytologically confirmed diagnosis
of HR+ HER2- advanced or metastatic breast cancer that is not amenable to curative
therapy and must have been previously treated with GnRH agonist at least 4 weeks prior
to start of study drug
• [For Part 1 and Part 2]: Had previous treatment for advanced or metastatic breast
cancer with:
1. ≤1 line of chemotherapy,
2. ≥1 cyclin-dependent kinases (CDK) 4/6 inhibitor, and
3. ≥1 antiestrogen therapy including, but not limited to, selective
estrogen-receptor degraders (eg, fulvestrant), selective estrogen receptor
modulators (eg, tamoxifen), and aromatase inhibitors (AI) (letrozole,
anastrozole, exemestane), and
4. ≥1 PARP inhibitor, if appropriate, if documented germline BRCA1/2 mutation Note:
Systemic local, loco-regional, or adjuvant treatment is not to be included in
enumeration or previous treatment
[For RLY-2608 + fulvestrant arm; Part 2, Group 2]: Received prior treatment with a PI3Kα
inhibitor and discontinued the inhibitor due to intolerance and not disease progression,
where intolerance is defined as treatment discontinuation due to treatment related AE (eg.
hyperglycemia, rash, diarrhea, stomatitis) other than severe hypersensitivity reaction
and/or life-threatening reactions, such as anaphylaxis and Stevens-Johnson syndrome.
Key Exclusion Criteria
Prior treatment with PI3Kα, AKT, or mTOR inhibitors (except for RLY-2608 + fulvestrant arm,
Part 2, Group 2).
Type 1 or Type 2 diabetes requiring antihyperglycemic medication, or fasting plasma glucose
≥140 mg/dL and glycosylated hemoglobin (HbA1c) ≥7.0%.
History of hypersensitivity to PI3K inhibitors. For combination arms only: hypersensitivity
to fulvestrant, palbociclib, and/or ribociclib, as appropriate for the combination.
For triple combination arms only: history of pneumonitis or interstitial lung disease.
For the single agent and combination arms other than with ribociclib: mean QT interval
corrected using Fridericia's formula (QTcF) >480 msec. For the combination arms with
ribociclib: mean QTcF ≥450 msec.
Patient has a history of prolonged QT syndrome or torsades de pointes. Patient has a
familial history of prolonged QT syndrome.
Clinically significant, uncontrolled cardiovascular disease CNS metastases or primary CNS
tumor that is associated with progressive neurologic symptoms
PIK3CA Mutation, Solid Tumor, Adult, HER2-negative Breast Cancer, Breast Cancer, Metastatic Breast Cancer, Advanced Breast Cancer, Unresectable Solid Tumor, Lip, Oral Cavity and Pharynx, Esophagus, Stomach, Small Intestine, Colon, Rectum, Anus, Liver, Pancreas, Other Digestive Organ, Larynx, Lung, Other Respiratory and Intrathoracic Organs, Bones and Joints, Soft Tissue, Kaposi's Sarcoma, Other Skin, Breast, Cervix, Corpus Uteri, Ovary, Other Female Genital, Prostate, Other Male Genital, Urinary Bladder, Kidney, Other Urinary, Eye and Orbit, Brain and Nervous System, Thyroid, Other Endocrine System, Unknown Sites, Ill-Defined Sites, Anal, Bladder, Brain/Central Nervous System, Colon and Rectum, Endocrine cancers, Gastrointestinal cancers, other, Genitourinary cancers, other, Head and Neck, Melanoma/Skin cancer, Sarcoma, Uterus
This study compares outcomes of prospective mesh-based breast reconstructions to historical
control breast reconstructions with no mesh.
Ahmed Afifi
Female
22 Years to 70 Years old
N/A
This study is NOT accepting healthy volunteers
NCT04646057
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Inclusion Criteria:
• Female between the inclusive ages of 22 and 70 at the time of initial expander surgery
• Scheduled to undergo either unilateral or bilateral mastectomy with immediate 2-stage
breast reconstruction
• Is able to understand the study requirements and is willing to provide written
informed consent
• Is willing and able to return for all scheduled study visits
Exclusion Criteria:
• Is pregnant or planning to become pregnant during study participation
• Has a history of failed tissue expansion or breast implantation at the intended
reconstruction site
• has a residual gross tumor at the intended reconstruction site
• has been treated for a systemic infection or a local infection at the surgical site
that the investigator determines will affect the safety of the subject during breast
reconstruction and/or mesh use
• has, as determined by the investigator, unsuitable tissue integrity for immediate
2-stage breast reconstruction
• has undergone previous radiation therapy to the reconstruction site or chest wall
• is scheduled to undergo post-operative radiation therapy at the reconstruction site
• has a Body Mass Index (BMI) < 14 or > 44
• has used nicotine products within 90 days of screening
• is currently taking medications including non-NSAID anti-coagulants,
immunosuppressants (including systemic steroids), or other medications determined by
the investigator to place the subject at an increased risk of local complications of
breast reconstruction
• has been diagnosed with a comorbid condition determined by the investigator to place
the subject at an increased risk of complications
• has participated in any other clinical study that the investigator feels may interfere
with this clinical study
Study of Dato-Dxd as Monotherapy and in Combination With Anti-cancer Agents in Patients With Advanced Solid Tumours (TROPION-PanTumor03)
TROPION-PanTumor03 will investigate the safety, tolerability, and anti-tumour activity of
Datopotamab Deruxtecan (Dato-DXd) as Monotherapy and in Combination with Anticancer Agents in
Patients with Advanced/Metastatic Solid Tumours.
Joshua Lang, Post Grad
All
18 Years to 130 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT05489211
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Key
Inclusion Criteria:
• Male and female, ≥ 18 years
• Documented advanced or metastatic malignancy
• Eastern Cooperative Oncology Group performance status of 0 or 1 with no deterioration
over the 2 weeks prior to baseline or day of first dosing
• All participants must provide a tumour sample for tissue-based analysis
• At least 1 measurable lesion not previously irradiated, except Substudy 3 (Prostate
Cancer) which allows participants with non measurable bone metastatic disease
• Adequate bone marrow reserve and organ function
• Minimum life expectancy of 12 weeks
• At the time of screening, contraceptive use by men or women should be consistent with
local regulations regarding the methods of contraception for those participating in
clinical studies
• All women of childbearing potential must have a negative serum pregnancy test
documented during screening
• Female participants must be 1 year post-menopausal, surgically sterile, or using 1
highly effective form of birth control. Female participants must not donate, or
retrieve for their own use, ova at any time during this study
• Male participants who intend to be sexually active with a female partner of
childbearing potential must be surgically sterile, avoid intercourse, or use a highly
effective method of contraception. Male participants must not freeze or donate sperm
at any time during this study.
• Capable of giving signed informed consent
• Provision of signed and dated written optional genetic research informed consent prior
to collection of samples for optional genetic research that supports the Genomic
Initiative
Key
Exclusion Criteria:
• Any evidence of diseases which, in the investigator's opinion, makes it undesirable
for the participant to participate in the study or that would jeopardize compliance
with the protocol
• History of another primary malignancy except for adequately resected basal cell
carcinoma or in situ squamous cell carcinoma of the skin, or other solid malignancy
treated with curative intent
• Persistent toxicities caused by previous anticancer therapy, excluding alopecia, not
yet improved
• Spinal cord compression or brain metastases unless treated
• Leptomeningeal carcinomatosis
• Clinically significant corneal disease
• Active hepatitis or uncontrolled hepatitis B or C virus infection
• Uncontrolled infection requiring IV antibiotics, antivirals or antifungals, for
example prodromal symptoms
• Known HIV infection that is not well controlled
• Active TB infection
• Significant cardiac diseases
• History of non-infectious Interstitial lung disease (ILD)/pneumonitis that required
steroids
• Has severe pulmonary function compromise
• Prior exposure to chloroquine/hydroxychloroquine without an adequate treatment washout
period
• Receipt of live, attenuated vaccine within 30 days prior to the first dose of study
intervention
• Prior exposure to anticancer therapies without an adequate treatment washout period
prior to enrolment or any concurrent anticancer treatment
• Major surgical procedure or significant traumatic injury within ≤ 3 weeks of the first
dose of study intervention or an anticipated need for major surgery during the study
• Prior treatment with TROP2-directed Anti-drug antibody, ADC Antibody-drug conjugate
(ADCs), other ADCs with deruxtecan payload
• Severe hypersensitivity to monoclonal antibodies
• Pregnant, breastfeeding, planning to become pregnant
Endometrial Cancer, Gastric Cancer, Metastatic Castration-resistant Prostate Cancer, Ovarian Cancer, Colorectal Cancer, Urothelial Cancer, Biliary Tract Cancer, Esophagus, Stomach, Colon, Rectum, Other Digestive Organ, Corpus Uteri, Ovary, Other Female Genital, Prostate, Other Male Genital, Colon and Rectum, Gastrointestinal cancers, other, Genitourinary cancers, other, Uterus
Trial of Therapeutic Hypothermia in Patients With ARDS (CHILL)
Acute Respiratory Distress Syndrome (ARDS) is a serious condition that occurs as a
complication of medical and surgical diseases, has a mortality of ~40%, and has no known
treatment other than optimization of support. Data from basic research, animal models, and
retrospective studies, case series, and small prospective studies suggest that therapeutic
hypothermia (TH) similar to that used for cardiac arrest may be lung protective in patients
with ARDS; however, shivering is a major complication of TH, often requiring paralysis with
neuromuscular blocking agents (NMBA) to control. Since the recently completed NHLBI PETAL
ROSE trial showed that NMBA had no effect (good or bad) in patients with moderate to severe
ARDS, the CHILL trial is designed to evaluate whether TH combined with NMBA is beneficial in
patients with ARDS. This Phase IIb randomized clinical trial is funded by the Department of
Defense to compare TH (core temperature 34-35°C) + NMBA for 48h vs. usual temperature
management in patients in 14 clinical centers with the Clinical Coordination Center and Data
Coordinating Center at University of Maryland Baltimore. Planned enrollment is 340 over ~3.5
years of the 4-year contract. COVID-19 is considered an ARDS risk-factor and patients with
ARDS secondary to COVID-19 pneumonia will be eligible for enrollment. Primary outcome is
28-day ventilator-free days. Secondary outcomes include safety, physiologic measures,
mortality, hospital and ICU length of stay, and serum biomarkers collected at baseline and on
days 1, 2, 3, 4, and 7.
Majid Afshar
All
18 Years to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04545424
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Inclusion Criteria:
1. endotracheal tube or tracheostomy in place and mechanically ventilated for ≤7 days;
2. admitted to a participating ICU
3. radiologic evidence of bilateral pulmonary infiltrates not fully explained by pleural
effusions, atelectasis, or hydrostatic pulmonary edema
4. P/F ratio ≤200 with PEEP ≥8 cm H2O; If ABG values are not available, the P/F ratio may
be inferred from SpO2 values based on Table 3 from Brown et al as long as following
conditions are met:
1. SpO2 values are 80-96%
2. SpO2 is measured ≥10 min after any change in FIO2
3. PEEP is ≥ 8 cm H2O
4. the pulse oximeter waveform tracing is adequate
5. the qualifying inferred P/F ratio is confirmed 1-6h after initial determination.
5. access to an LAR to provide consent.
6. Criteria 3 AND 4 must be met within 72h of enrollment and randomization, not be fully
explained by hydrostatic pulmonary edema, and must have occurred within 7 days of
exposure to an ARDS-risk factor (including continuous exposure to persistent processes
(e.g. sepsis, pneumonia, COVID-19).
• Patients may be enrolled and decision about randomization delayed if all criteria
other than P/F ratio ≤ 200 are met and then randomized if and when the P/F ratio
≤200 (as long as this occurs within 72h of randomization). Patients on high flow
nasal oxygen or non-invasive pressure ventilation may be consented if they meet
criteria for starting the 72h ARDS window but may not be enrolled and randomized
until they are intubated.
Exclusion Criteria:
1. Missed moderate-severe ARDS window (>72hrs) •Window starts when patient is intubated
with a qualifying P/F ratio of ≤ 200 with PEEP ≥ 8 cm H2O or on high flow nasal oxygen
with well-fitting nasal cannula with flow ≥ 40 LPM and FiO2 ≥ 0.65 or on non-invasive
pressure ventilation with PEEP ≥ 8 cm H2O and FiO2 ≥ 0.6.
2. Missed NMB window: (>48 hrs)
3. Missed mechanical ventilation window (>7 days)
4. Refractory hypotension (continuous infusion of >0.3 mcg/kg/min of norepinephrine or
equivalent dose of other vasopressors within 2 hours prior to randomization)
5. Core temperature <35.5°C for ≥6 hours while not receiving CRRT on day of randomization
6. Significant, active bleeding (>3u blood products and/or surgical/IR intervention) on
day of randomization
7. Platelets <10K/mm3 (uncorrected) on day of randomization
8. Active hematologic malignancy
9. Skin process that precludes cooling device
10. Moribund, not likely to survive 72h
11. Pre-morbid condition makes it unlikely that patient will survive 28 days
12. Do Not Resuscitate status at time of randomization (excluding patients receiving full
support EXCEPT CPR for cardiac arrest)
13. Not likely to remain intubated for ≥48h
14. Physician of record unwilling to participate
15. Severe underlying lung disease
1. Needs > 2 LPM or >28% continuous home O2 (adjusted for altitude
2. On BIPAP (except for OSA)
3. Prior lung transplantation
16. Pregnant at time of randomization
17. BMI consistently >50 kg/m2
18. Known NYHA class IV heart disease
19. Acute Coronary Syndrome (MI, unstable angina) within 30 days of randomization
20. Cardiac arrest within 30 days of randomization
21. Burns over >20% of the body surface
22. Severe chronic liver disease (Child-Pugh score 12-15)
23. Previously randomized in CHILL study
24. Simultaneous enrollment in another inpatient interventional trial started during the
current hospitalization.
25. On ECMO during the current hospitalization.
An Open-label Study of Ozanimod in Moderate to Severe Ulcerative Colitis in Clinical Practice
The purpose of this study is to explore the safety, efficacy, effects on quality of life
(QOL), and biomarker response of ozanimod in participants with moderate to severely active
ulcerative colitis (UC) in clinical practice.
Freddy Caldera
All
18 Years and over
Phase 4
This study is NOT accepting healthy volunteers
NCT05369832
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Inclusion Criteria:
• A diagnosis of ulcerative colitis (UC), with signs and symptoms consistent with UC for
at least 3 months prior to the first study intervention administration
• Moderate to severely active UC disease activity, defined as a modified Mayo score of 4
through 9, inclusive, with the following minimum subscores:
i) An SF subscore ≥ 1, AND ii) An RB subscore ≥ 1, AND iii) An ES ≥ 2 (endoscopy
performed within 60 days of the first study intervention administration).
• Report of a previous colonoscopy that documents extent of disease
Exclusion Criteria:
• Current or recent (within 3 months of screening) evidence of fulminant colitis, toxic
megacolon, or bowel perforation
• Extensive colonic resection or current stoma
• Colonic dysplasia that has not been removed
Other protocol-defined inclusion/exclusion criteria apply
Phase I Trial to Evaluate VLP Peanut in Healthy and Peanut Allergic Subjects (PROTECT)
This phase I clinical trial is designed to evaluate the safety and tolerability of VLP Peanut
in healthy subjects and in subjects with peanut allergy (PA). This clinical trial will
evaluate the immunotoxicity profile of VLP Peanut in healthy subjects and assess the
immunotoxicity profile and the degree of reactogenicity (allergenicity) in subjects with PA.
This clinical trial will also explore preliminary proof of efficacy of VLP Peanut in subjects
with PA.
Mark Moss
All
18 Years to 50 Years old
Phase 1
This study is also accepting healthy volunteers
NCT05476497
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Part A Main
Inclusion Criteria:
1. Capable of giving signed informed consent.
2. Subject who has a signed and dated Informed Consent Form (ICF).
3. Subject must be 18 to 50 years inclusive, at the time of signing the ICF.
4. Male or female.
5. Female subjects who are not of childbearing potential (or females of childbearing
potential who agree to comply with the contraceptive requirements of the clinical
trial protocol).
6. Good general health, as determined by the Investigator.
7. A positive SPT to histamine.
The following additional inclusion criteria are only applicable to the healthy
subjects in Group A1:
8. Healthy subjects (non-atopic) with no clinically significant co-morbidity (including
broncho-reactive airway disease like asthma, current allergic rhinitis, etc.).
9. Subjects with no history of allergy or intolerance to peanut or any other food and who
consume peanuts with no effect.
10. Subjects with lack of sensitivity to peanut allergen confirmed by SPT using whole
peanut extract.
11. Peanut specific immunoglobulin E (IgE) <0.1 kU/L.
12. Ara h 2 specific IgE <0.1 kU/L.
13. Subjects with negative basophil activation test (BAT).
The following additional inclusion criteria are only applicable to the subjects with
PA in Group A2:
14. Clinical history of physician diagnosed PA.
15. Peanut allergen sensitivity confirmed by SPT and IgE.
16. Subjects who currently adhere to a strict peanut-free diet and who agree to continue
this for the duration of the clinical trial.
17. Subjects who are able to handle and correctly use an adrenaline auto-injector.
Part B Main
Inclusion Criteria:
1. Capable of giving signed informed consent.
2. Subject who has a signed and dated ICF.
3. Subjects aged 18 to 50 years of age inclusive, at the time of signing the ICF.
4. Male or female.
5. Female subjects who are not of childbearing potential (or females of childbearing
potential who agree to comply with the contraceptive requirements of the clinical
trial protocol).
6. Clinical history of physician diagnosed PA.
7. Peanut allergen sensitivity confirmed by SPT and IgE (Peanut specific IgE ≥5.0 kU/L
and Ara h 2 specific IgE ≥2.0 kU/L)
8. Subjects with positive BAT.
9. Subjects who currently adhere to a strict peanut-free diet and who agree to continue
this for the duration of the clinical trial.
10. Good general health, as determined by the Investigator.
11. Subjects who are able to handle and correctly use an adrenaline auto-injector.
Main Exclusion Criteria Part A and B:
1. Pregnant or lactating subject.
2. Presence of any medical condition that may reduce the ability to survive a serious
allergic reaction.
3. Subjects with atopic dermatitis with >25% skin surface involvement.
4. For subjects with PA, presence of severe, poorly controlled or uncontrolled asthma.
5. History of severe or life-threatening anaphylactic reactions to peanut resulting in
neurological compromise or requiring mechanical ventilation.
6. Clinical history of severe systemic or life-threatening anaphylactic reactions to
other foods (apart from peanut), insect venom, exercise, drugs, etc. or idiopathic
anaphylaxis.
7. Unable to receive epinephrine therapy or at greater risk of developing adverse
reactions after epinephrine administration as assessed by the site Investigator.
8. Clinical history of drug or alcohol abuse, which, in the Investigator's opinion, could
interfere with the subject's ability to participate in the clinical trial.
9. Participation in a clinical research trial with any investigational drug/placebo
within 3 months of screening (Visit 1) or concomitantly with this clinical trial.
10. Personal, financial or other dependent relationship (e.g., employee or immediate
relative) with the clinical trial site, Sponsor, Sponsor's representative, or another
individual who has access to the clinical trial protocol.
11. Vulnerable subjects or those in judicial or governmental detention, detainment or
imprisonment in a public institution.
Peanut Allergy, Allergic rhinitis due to food, Other, Infections, Immune System & Allergies
Using Nivolumab Alone or With Cabozantinib to Prevent Mucosal Melanoma Return After Surgery
This phase II trial tests whether nivolumab in combination with cabozantinib works in
patients with mucosal melanoma. Immunotherapy with monoclonal antibodies, such as nivolumab,
may help the body's immune system attack the cancer, and may interfere with the ability of
tumor cells to grow and spread. Cabozantinib may stop the growth of tumor cells by blocking
some of the enzymes needed for cell growth. It works by blocking the action of an abnormal
protein that signals tumor cells to multiply. This helps stop the spread of tumor cells.
Giving nivolumab in combination with cabozantinib could prevent cancer from returning.
Vincent Ma, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05111574
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Inclusion Criteria:
• STEP 0 INCLUSION CRITERIA
• Histologically proven mucosal melanoma by local pathology
• Central PD-L1 tumor tissue submission
• STEP 1 INCLUSION CRITERIA
• Receipt of the central PD-L1 testing results available
• Disease status-Resected R0 or R1 disease patients. Patients eligible for randomization
have resected R0 or R1 disease (with negative margins or positive microscopic margins)
that must meet one of the following 4 criteria as defined below:
• Regional lymph node (LN) involvement; OR
• In-transit metastases/satellite primary disease; OR
• Single localized, primary disease meeting one of the following site-specific
requirements:
• Head/neck •Sinonasal (including nasopharynx): any primary lesion; Nasal or
oral cavity; pT4a or above, given slightly improved OS
• NOTE: Conjunctival: does not meet the qualification for eligibility
• Anorectal •any primary lesion
• Vaginal/cervical •any primary, as they have 5 year OS rates of 5-25
• Urinary tract •any primary urethral or bladder tumor
• Penile
• Vulvar- AJCC cutaneous stage IIB or higher
• Esophageal/gallbladder •any primary
• Locoregionally recurrent following prior resection, meeting at least one of the
above criteria
• In addition, patients must have undergone cross-sectional imaging of the brain,
chest, abdomen and pelvis with no evidence of distant metastatic disease
• Disease status-Non-resected R2 or metastatic disease patients
• Non-resected R2 or metastatic disease that is assessable and measurable
radiographically or by physical examination
• Prior Treatment:
• No prior systemic checkpoint inhibitor therapy of mucosal melanoma, including in
the adjuvant setting, is allowed. Prior adjuvant chemotherapy or interferon is
allowed.
• No other active, concurrent malignancy that requires ongoing systemic treatment
or interferes with radiographic assessment of melanoma response as determined by
the investigator. Exceptions may allow for adjuvant no evidence of disease (NED)
cancers undergoing hormone based therapy may be eligible pending the other
eligibility criteria are met and the principal investigator (PI) affirms the
hormonal agent would not change the melanoma response.
• Any radiation must have completed 28 days prior to randomization and the patient
must have adequately recovered from its effects.
• For resectable patients only: Surgery must have completed 28 days prior to
randomization.
• For resectable patients only: Surgery must have completed no more than 84 days
prior to randomization.
• Not pregnant and not nursing, because this study has an agent that has known
genotoxic, mutagenic and teratogenic effects. Therefore, for women of childbearing
potential only, a negative pregnancy test done =< 7 days prior to registration is
required
• Age >= 18 years
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2
• Absolute neutrophil count (ANC) >= 1,500/mm^3
• Platelet count >= 100,000/mm^3
• Creatinine =< 1.5 x upper limit of normal (ULN) OR creatinine clearance (CrCl) >=
50mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
• Albumin >= 2.8 g/dL
• Total bilirubin =< 1.5 x upper limit of normal (ULN)
• Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =< 2.5 x upper limit
of normal (ULN)
• No cardiovascular disease, including:
• No history of acute coronary syndromes (including myocardial infarction and
unstable angina), coronary artery bypass graft (CABG) coronary angioplasty, or
stenting within 6 months prior to study entry.
• No history of current class II or higher congestive heart failure as defined by
the New York Heart Association (NYHA) functional classification system.
• No refractory hypertension defined as a blood pressure of systolic > 140 mmHg
and/or diastolic > 90 mmHg despite adequate attempts at anti-hypertensive
therapy.
• No history of myocarditis.
• No history of syncope of cardiovascular etiology, uncontrolled cardiac
arrhythmia, history of Mobitz II second degree or third degree heart block
without a permanent pacemaker in Association (NYHA) class II to IV heart failure,
or stroke/transient ischemic attack (TIA) within the past 3 months.
• No corrected QT interval by Fridericia's formula (QTcF) > 500 msec. Note: if
initial QTcF is found to be > 500 ms, two additional EKGs separated by at least 3
minutes should be performed. If the average of these three consecutive results
for QTcF is =< 500 ms, the subject meets eligibility in this regard.
• No underlying hematologic issues, including:
• Congenital bleeding diathesis
• Gastrointestinal (GI) bleeding requiring intervention within the past 6 months,
unless directly related to mucosal melanoma
• Active hemoptysis within 42 days prior to study enrollment.
• Active tumor lesions with cavitations or tumor lesions which invade, encase, or
abut major blood vessels. The anatomic location and characteristics of primary
tumors or metastases as well as the medical history should be carefully reviewed
in the selection of subjects for treatment with cabozantinib/placebo.
• Pulmonary emboli or deep vein thromboses (DVT) that require an active
anticoagulation regimen.
• No known or suspected history of cytopenia (low white blood cell [WBC],
hemoglobin or platelet count) of greater than 3 months duration with an unknown
cause, myelodysplastic syndrome, or hematologic malignancies.
• No clinical, laboratory or radiographic evidence of an active bacterial, fungal, or
viral infection requiring treatment at the time of pre-registration (e.g., active
symptoms of COVID-19 infection or a post-infectious symptomatic autoimmune syndrome,
serious bacterial infections requiring antibiotics).
• No known or suspected gastrointestinal disorder affecting absorption of oral
medications.
• Comorbid conditions:
• No active autoimmune disease or any condition requiring systemic treatment with
either corticosteroids (> 10 mg daily of prednisone equivalents) or other
immunosuppressive medications within 14 days of study drug administration.
Inhaled or topical steroids and adrenal replacement doses > 10 mg daily
prednisone equivalents are permitted in the absence of active autoimmune disease.
• No history of autoimmune motor neuropathy (e.g., Guillain-Barre syndrome,
myasthenia gravis) or non-infectious pneumonitis.
• No history of severe allergic reactions to an unknown allergen or any components
of the study drugs or its excipients.
• No history of gastrointestinal perforation or abdominal fistula.
• No clinically suspected central nervous system (CNS) (leptomeningeal or
parenchymal) metastases. Patients with a history of CNS metastasis(s) will be
allowed as long as
• The metastatic site(s) were adequately treated as demonstrated by clinical
and radiographic improvement, AND
• The patient has recovered from the intervention (no residual adverse events
> Common Terminology Criteria for Adverse Events [CTCAE] grade 1), AND
• The patient has remained without occurrence of new or worsening CNS symptoms
for a period of 28 days prior to enrollment.
• No history of seizure or any condition that may increase the patient's seizure
risk (e.g., prior cortical stroke, significant brain trauma) within 2 years.
• No clinically active or chronic liver disease resulting in moderate/severe
hepatic impairment (Child-Pugh class B or C), ascites, coagulopathy or bleeding
due to liver dysfunction.
• No untreated spinal cord compression or evidence of spinal metastases with a risk
of impending fracture or spinal cord compression. Spinal metastases must have
completed planned radiation or surgical therapy prior to registration.
• Concomitant medications:
• Chronic concomitant treatment with strong inhibitors of CYP3A4 is not allowed on
this study. Patients on strong CYP3A4 inhibitors must discontinue the drug for 5
days prior to the start of study treatment.
• Chronic concomitant treatment with strong CYP3A4 inducers is not allowed.
Patients must discontinue the drug 5 days prior to the start of study treatment.
Melanoma, Skin, Melanoma/Skin cancer, Anal Melanoma, Bladder Melanoma, Cervical Melanoma, Esophageal Melanoma, Gallbladder Melanoma, Mucosal Melanoma, Mucosal Melanoma of the Head and Neck, Mucosal Melanoma of the Urinary System, Nasopharyngeal Melanoma, Oral Cavity Mucosal Melanoma, Penile Mucosal Melanoma, Rectal Melanoma, Recurrent Mucosal Melanoma, Sinonasal Mucosal Melanoma, Stage II Vulvar Cancer AJCC v8, Stage III Vulvar Cancer AJCC v8, Stage IV Vulvar Cancer AJCC v8, Urethral Melanoma, Vaginal Melanoma, Vulvar Melanoma
A Study Evaluating the Efficacy and Safety of IV L-Citrulline for the Prevention of Clinical Sequelae of Acute Lung Injury Induced by Cardiopulmonary Bypass in Pediatric Patients Undergoing Surgery for Congenital Heart Defects
This is a randomized, double-blind, placebo controlled, multicenter study to compare the
efficacy and safety of L-citrulline versus placebo in patients undergoing surgery for
congenital heart defects. Eligible patients undergoing repair of a large unrestrictive
ventricular septal defect (VSD), a partial or complete atrioventricular septal defect (AVSD),
or an ostium primum atrial septal defect (primum ASD) will be eligible for enrollment.
Petros Anagnostopoulos
All
up to 18 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05253209
Show full eligibility criteria
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Inclusion Criteria:
• Patients, parents, or legal guardian willing and able to sign informed consent
• Male and female subjects aged ≤18 years of age (females of child-bearing potential
willing to practice an acceptable form of birth control)
• Patients undergoing cardiopulmonary bypass for repair of a large unrestrictive
ventricular septal defect, an ostium primum/secundum atrial septal defect, or a
partial or complete atrioventricular septal defect
• Pre-operative echocardiogram confirming cardiovascular anatomy and defect to be
repaired
Exclusion Criteria:
• Evidence of pulmonary artery or vein abnormalities that will not be addressed
surgically. Specific abnormalities excluded include:
• significant pulmonary artery narrowing not amenable to surgical correction
• previous pulmonary artery stent placement
• significant left sided AV valve regurgitation not amenable to surgical correction
• pulmonary venous return abnormalities not amenable to surgical correction
• pulmonary vein stenosis not amenable to surgical correction
• Preoperative requirement for mechanical ventilation or IV inotrope support
• Presence of fixed or idiopathic pulmonary hypertension (i.e. Eisenmenger's Syndrome)
prior to surgical repair
• Pre-operative use of medications to treat pulmonary hypertension
• Pregnancy; Sexually active females of child-bearing potential must be willing to
practice an acceptable method of birth control for the duration of study participation
(e.g. oral contraceptive, hormonal implant, intra-uterine device)
• Participation in another clinical trial within 30 days of Screening or while
participating in the current study, including the 28 days of follow-up post study drug
administration.
• Any condition which, in the opinion of the investigator, might interfere with the
study objectives
A Study of FF-10850 Topotecan Liposome Injection in Advanced Solid Tumors
To determine the safety profile, maximum tolerated dose (MTD), dose-limiting toxicities
(DLTs), and recommended Phase 2 dose (RP2D) of FF-10850 (topotecan liposome injection) in
patients with advanced solid tumors.
Vincent Ma, MD
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04047251
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Inclusion Criteria:
Patients must meet all the following criteria to participate in the study:
1. Males and females ≥ 18 years of age
2. Dose-escalation phase: Histologically or cytologically confirmed metastatic and/or
unresectable solid tumor, relapsed or refractory to standard therapy, or for which no
standard therapy is available that is expected to improve survival by at least 3
months
3. At least 3 weeks beyond the last chemotherapy (or 3 half-lives, whichever is shorter),
radiotherapy, major surgery, or experimental treatment, and recovered from all acute
toxicities (≤ Grade 1), prior to the first dose of FF-10850
4. Adequate performance status: Eastern Cooperative Oncology Group (ECOG) ≤ 1
5. Life expectancy of ≥ 3 months
6. Adequate hematologic parameters without ongoing transfusion support:
• Hemoglobin (Hb) ≥ 9 g/dL
• Absolute neutrophil count (ANC) ≥ 1.0 × 109 cells/L
• Platelets ≥ 100 × 109 cells/L
7. Creatinine ≤ 1.5 × ULN, or calculated creatinine clearance ≥ 50 mL/minute by either
the Cockcroft-Gault formula or as measured by a 24-hour urine collection
8. Total bilirubin ≤ 2 × ULN unless due to Gilbert's disease; patients with Gilbert's
disease who have a total bilirubin > 6 mg/dL are to be excluded
9. ALT and AST ≤ 2.5 times ULN, or < 5 × ULN for patients with liver metastases
10. QT interval corrected for rate (QT interval corrected for rate using Fridericia's
Correction Formula, QTcF) ≤ 470 msec for women and ≤ 450 msec for men on the ECG
obtained at Screening and confirmed pre-treatment on Cycle 1 Day 1.
11. Patient must be willing to undergo a tumor biopsy, if the patient has a
biopsy-accessible tumor
Exclusion Criteria:
1. Patients who have not received standard/approved therapies expected to improve
survival by at least 3 months
2. History of severe hypersensitivity reactions to topotecan
3. Serious cardiac condition within the last 6 months, such as uncontrolled arrhythmia,
myocardial infarction, unstable angina or heart disease defined by the New York Heart
Association (NYHA) Class III or Class IV or hereditary long QT syndrome
4. Concomitant medication(s) that may cause QTc prolongation or induce Torsades de
Pointes, except for antimicrobials that are used as standard of care to prevent or
treat infections and other such drugs that are considered by the Investigator to be
essential for patient care
5. Active central nervous system (CNS) malignant disease in patients with a history of
CNS malignancy. Patients with previously treated stable brain metastases are allowed
if they have been stable off steroid therapy for at least 4 weeks.
6. Known positive for human immunodeficiency virus (HIV), hepatitis B virus surface
antigen (HBsAg) or hepatitis C virus (HCV)
7. Active infection requiring intravenous (IV) antibiotic usage within the last week
prior to study treatment
8. Any other medical intervention or other condition which, in the opinion of the
Principal Investigator, could compromise adherence to study requirements or confound
the interpretation of study results
9. Pregnant or breast-feeding
Other Skin, Melanoma/Skin cancer, Advanced Solid Tumors
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (Uni-Rare)
This is an international, multicenter study with two components:
Registry
- A standardized genetic screening and a prospective, standardized, cross-sectional
clinical data collection
- Enrollment is open to all genes on the RD Rare Gene List
Natural History Study
- A prospective, standardized, longitudinal Natural History Study
- Enrollment opens gene-by-gene, based on funding and within-gene Registry enrollment The
study objectives are as follows.
Registry Objectives
1. Genotype Characterization
2. Cross-Sectional Phenotype Characterization (within gene)
3. Establish a Link to My Retina Tracker Registry (MRTR)
4. Ancillary Exploratory Studies - Pooling of Genes
Natural History Study Objectives
1. Natural History (within gene)
2. Structure-Function Relationship (within gene)
3. Risk Factors for Progression (within gene)
4. Ancillary Exploratory Studies - Pooling of Genes
Kimberly Stepien, MD
All
4 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05589714
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Inclusion Criteria:
Participants must meet all the following inclusion criteria at the
Registry/Screening Visit to be eligible to enroll into the genetic screening phase:
1. Willing to participate in the study and able to communicate consent during the consent
process
2. Willing and able to complete all applicable Registry/Screening Visit assessments
3. Age ≥ 4 years
4. Must have a single gene on the RD Rare Gene List which meets one of the Genetic
Screening Criteria below based on a genetic report* from a clinically certified lab
(or from a research lab which has been approved by the study Genetics Committee):
Inheritance Pattern is Recessive and has at least 2 disease-causing variants which are
homozygous or heterozygous in trans
OR
Inheritance Pattern is Recessive and has 2 disease-causing variants with unknown phase and
meets all the following additional informatic criteria that is consistent with likely
segregation in trans:
1. Investigator confirms genotype and phenotype are consistent with autosomal recessive
inheritance
2. The 2 disease-causing variants have not been reported in cis in variant databases
3. No additional potentially pathogenic variants were found on the gene (and the
sequencing data for the gene were sufficiently robust to detect any additional
potentially pathogenic variants)
4. No potentially pathogenic variants were found in other common, likely candidate genes
for the proposed condition
OR
Inheritance Pattern is Dominant, X-linked, or Mitochondrial and has at least 1
disease-causing variant
Both eyes must meet the following criteria at the Registry/Screening Visit to enroll into
the genetic screening phase:
1. Both eyes must have a clinical diagnosis of retinal dystrophy
2. Both eyes must permit good quality photographic imaging (e.g., but not limited to,
clear ocular media, adequate pupil dilation, stable fixation)
Exclusion Criteria:
Participants must not meet any of the following exclusion criteria at the
Registry/Screening Visit to be eligible to enroll into the genetic screening phase:
1. History of more than 1 year of cumulative treatment, at any time, with an agent
associated with pigmentary retinopathy including amiodarone, chloroquine, deferoxamine,
hydroxychloroquine, pentosan polysulfate, tamoxifen, and deferoxamine Note: Since this is
an observational study, pregnant women will not be specifically excluded from
participation. However, minors that are pregnant shall be precluded from participation
until they become the age of majority.
Ocular
Exclusion Criteria:
If either eye has any of the following ocular exclusion criteria at the Registry/Screening
Visit, then the participant is not eligible to enroll into the genetic screening phase:
1. Current vitreous hemorrhage
2. Current complications of pathological myopia (for example, but not limited to, myopic
maculopathy including atrophy, scar, choroidal neovascularization, schisis) that could
inhibit ability to obtain good quality photographic imaging
3. History of intraocular surgery (for example, but not limited to, cataract surgery,
vitrectomy, penetrating keratoplasty, or LASIK) within 3 months of Registry/Screening
Visit
4. Current or any history of confirmed diagnosis of glaucoma (for example, but not
limited to, glaucomatous VF changes or nerve changes, or history of glaucoma filtering
surgery)
5. Current or any history of retinal vascular occlusion or proliferative diabetic
retinopathy
6. History or current evidence of ocular disease that, in the opinion of the
Investigator, may confound assessment of visual function (for example, but not limited
to, tractional or rhegmatogenous retinal detachment, any vitreoretinal surgery,
retinal vascular occlusion, proliferative diabetic retinopathy)
7. The following medications and treatments are prohibited as they can affect progression
of retinitis pigmentosa (RP). The participant must not have received the following
treatments:
Any use of ocular stem cell or gene therapy Any treatment with ocriplasmin Treatment
with Ozurdex (dexamethasone), Iluvien, or Yutiq (fluocinolone acetonide) intravitreal
implant
8. The following medications and treatments are excluded within the specified timeframe:
Treatment with an ophthalmic oligonucleotide within the last 9 months (last treatment date
is less than 9 months prior to Registry/Screening Visit date)
Treatment with any other product within five times the expected half-life of the product
(time from last treatment date to Registry/Screening Visit date is at least 5 times the
half-life of the given product)
Fluorescence-based Detection of Inflammation and Necrosis to Inform Surgical Decision-making and Enhance Outcomes
This study investigates fluorescence image-guided surgery to allow precise identification of
necrotic tissue both preoperatively and intraoperatively in burn patients. Furthermore, it
uses a multi-model approach to elucidate the localization of ICG in inflammation and necrosis
to determine how this novel use of a well-known fluorescence marker can be optimized to aid
in surgical decision making. This proposal will provide the necessary data to support the
design of a larger clinical trial to study the feasibility and efficacy of this technology to
improve the precision of necrosis detection and removal and improve wound healing outcomes.
Up to 100 participants will be on study for up to approximately 24 days.
Angela Gibson
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05593523
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Inclusion Criteria:
• English speaker
• Patients with partial thickness indeterminate depth burn wounds that occurred within
24 hours of admission and are expected to require admission for at least 3 days (Aim
1) or with deep partial thickness or full thickness burn wounds that are 1-30% TBSA
and will likely require surgery (Aim 2)
• Subject understands the study procedures and can provide informed consent to
participate in the study and authorization for release of relevant protected health
information to the study investigator
Exclusion Criteria:
• Contraindication to Indocyanine Green (ICG) injection, i.e. previous reaction to ICG
(adverse event rate: 1 in 42,000) or Iodine allergy.
• Inability to obtain consent
• Subject with pre-existing inflammatory diseases or chronically treated before
admission to the hospital with steroids or nonsteroidal anti-inflammatory drugs or
biologics
• Subject with immune deficiency (HIV infection or use of corticosteroids, cytostatic
drugs, tetracycline and certain bisphosphonates)
• Subject with known or suspected infections or on antibiotic therapy
• Subject known or suspected to be pregnant
Burn Wound, Burn of unspecified body region, unspecified degree, Other, Skin & Dermatology, Injury, Trauma & Emergency Medicine, Surgical Procedures
Testing the Addition of an Anti-cancer Drug, Ipatasertib, to the Usual Immunotherapy Treatment (Pembrolizumab) in Patients With Recurrent or Metastatic Squamous Cell Cancer of the Head and Neck
This phase II trial compares the effect of adding ipatasertib to pembrolizumab (standard
immunotherapy) vs. pembrolizumab alone in treating patients with squamous cell cancer of the
head and neck that has come back (recurrent) or that has spread from where it first started
(primary site) to other places in the body (metastatic). Ipatasertib is in a class of
medications called protein kinase B (AKT) inhibitors. It may stop the growth of tumor cells
and may kill them. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help
the body's immune system attack the cancer, and may interfere with the ability of tumor cells
to grow and spread. Giving ipatasertib in combination with pembrolizumab may be more
effective than pembrolizumab alone in improving some outcomes in patients with
recurrent/metastatic squamous cell cancer of the head and neck.
Justine Bruce, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05172258
Show full eligibility criteria
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Inclusion Criteria:
• Patients must have histologically or cytologically confirmed recurrent or metastatic
HNSCC that is considered incurable.
• Patients must have measurable disease, defined as at least one lesion that can be
accurately measured in at least one dimension (longest diameter to be recorded for
non-nodal lesions and short axis for nodal lesions) as >= 20 mm (>= 2 cm) by chest
x-ray or as >=10 mm (>= 1 cm) with CT scan, magnetic resonance imaging (MRI), or
calipers by clinical exam.
• Primary tumor locations of oral cavity, oropharynx, hypopharynx, and larynx are
allowed. Participants may not have a primary tumor site of nasopharynx.
• Patients with oropharyngeal cancer must have known human papillomavirus (HPV) status
defined by human papillomavirus type 16 (p16) testing.
• Patients should not have had prior systemic therapy administered in the recurrent or
metastatic setting. Systemic therapy which was given as part of multimodal treatment
for locally advanced disease is allowed.
• Patients must be able to provide an archival tissue specimen.
• Patients must be willing to undergo a mandatory tumor biopsy on treatment
• Tumor tissue must have a documented combined positive score (CPS) of >= 1 for PD-L1.
• Age >= 18 years. Because no dosing or adverse event data are currently available on
the use of ipatasertib in combination with pembrolizumab in patients < 18 years of
age, children are excluded from this study.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 (Karnofsky >=
60%).
• Absolute neutrophil count >= 1,000/mcL.
• Platelets >= 100,000/mcL.
• Total bilirubin =< 1.5 x institutional upper limit of normal (ULN).
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase
[SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT])
=< 3 x institutional ULN.
• Creatinine =< 1.5 x institutional ULN.
• Glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2 (using the Cockcroft-Gault
formula).
• Human immunodeficiency virus (HIV) infected patients on effective antiretroviral
therapy with undetectable viral load within 6 months are eligible for this trial.
• For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral
load must be undetectable on suppressive therapy, if indicated.
• Patients with a history of hepatitis C virus (HCV) infection must have been treated
and cured. For patients with HCV infection who are currently on treatment, they are
eligible if they have an undetectable HCV viral load.
• Patients with treated brain metastases are eligible if follow-up brain imaging after
central nervous system (CNS)-directed therapy shows no evidence of progression.
• Patients with a prior or concurrent malignancy whose natural history or treatment does
not have the potential to interfere with the safety or efficacy assessment of the
investigational regimen are eligible for this trial.
• Patients with known history or current symptoms of cardiac disease, or history of
treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac
function using the New York Heart Association Functional Classification. To be
eligible for this trial, patients should be class 2B or better.
• Patients must be able to swallow orally administered medication whole.
• For women of childbearing potential (WOCBP): agreement to remain abstinent (refrain
from heterosexual intercourse) or use contraceptive methods with a failure rate of <
1% per year during the treatment period, for 5 months after the last dose of
pembrolizumab and 28 days after the last dose of ipatasertib, and agreement to refrain
from donating eggs during this same period.
• For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use
contraceptive measures, agreement to refrain from donating sperm during the treatment
period, and for 5 months after the last dose of pembrolizumab and 28 days after the
last dose of ipatasertib.
• Ability to understand and the willingness to sign a written informed consent document.
Participants with impaired decision-making capacity (IDMC) who have a
legally-authorized representative (LAR) and/or family member available will also be
eligible.
Exclusion Criteria:
• Prior treatment with a checkpoint inhibitor given for relapsed or metastatic disease.
Prior treatment with a checkpoint inhibitor for locally advanced disease as part
multidisciplinary treatment is allowed.
• History of malabsorption syndrome or other condition that would interfere with enteral
absorption or result in the inability or unwillingness to swallow pills.
• Patient has a diagnosis of immunodeficiency or is receiving systemic steroid therapy
(in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of
immunosuppressive therapy within 7 days prior to the first dose of trial treatment.
The use of physiologic doses of corticosteroids may be approved after consultation
with the study principal investigator (PI).
• Active autoimmune disease that has required systemic treatment in the past 2 years
(i.e. with use of disease modifying agents, corticosteroids, or immunosuppressive
drugs). Replacement therapy (e.g., thyroxine or physiologic corticosteroid replacement
for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic
treatment.
• Type 1 or Type 2 diabetes mellitus requiring insulin at study entry are ineligible.
• Patients who are on a stable dose of oral diabetes medication >= 4 weeks prior to
initiation of study treatment may be eligible for enrollment. Patients must meet
the laboratory eligibility criteria for fasting blood glucose and hemoglobin A1c.
• Fasting glucose =< 150 mg/dL (8.3 mmol/L) and hemoglobin A1c =< 7.5% (58
mmol/mol).
• Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for
nitrosoureas or mitomycin C) prior to entering the study.
• Patients who have not recovered from adverse events due to prior anti-cancer therapy
(i.e., have residual toxicities > grade 1). Note: Patients with grade =< 2 neuropathy
or grade =< 2 alopecia are an exception to this criterion and may qualify for the
study. Note: If patients received major surgery, they must have recovered adequately
from the toxicity and/or complications from the intervention prior to starting
therapy.
• Patients who are receiving any other investigational agents.
• Patients with known active CNS metastases and/or carcinomatous meningitis. Patients
with previously treated brain metastases may participate provided they are
radiologically stable, i.e., without evidence of progression for at least 4 weeks by
repeat imaging (note that the repeat imaging should be performed during study
screening), clinically stable and without requirement of steroid treatment for at
least 14 days prior to first dose of study treatment.
• History of allergic reactions attributed to compounds of similar chemical or biologic
composition to ipatasertib or hypersensitivity (grade >= 3) to pembrolizumab and/or
any of the components of the solution for injection.
• Treatment with strong CYP3A inhibitors or strong CYP3A inducers within 2 weeks or 5
drug-elimination half-lives, whichever is longer, prior to initiation of study drug is
prohibited. Because the lists of these agents are constantly changing, it is important
to regularly consult a frequently-updated medical reference. As part of the
enrollment/informed consent procedures, the patient will be counseled on the risk of
interactions with other agents, and what to do if new medications need to be
prescribed or if the patient is considering a new over-the-counter medicine or herbal
product.
• Patients with uncontrolled intercurrent illness (including but not limited to
interstitial lung disease or active, non-infectious pneumonitis) or a history of
(non-infectious) pneumonitis that required steroids.
• Patients with psychiatric illness/social situations that would limit compliance with
study requirements.
• Patients who are pregnant or breastfeeding, or are expecting to conceive or father
children within the projected duration of the study, starting with the screening visit
through 5 months after the last dose of study treatment. A WOCBP who has a positive
urine pregnancy test (e.g., within 72 hours) prior to treatment will be excluded from
the study. If the urine test is positive or cannot be confirmed as negative, a serum
pregnancy test will be required. Pregnant women are excluded from this study because
pembrolizumab is a monoclonal antibody agent and ipatasertib is an oral AKT inhibitor
with the potential for teratogenic or abortifacient effects. Because there is an
unknown but potential risk for adverse events in nursing infants secondary to
treatment of the mother with pembrolizumab and ipatasertib, breastfeeding should be
discontinued if the mother is treated with pembrolizumab or ipatasertib. Due to the
potential risks, WOCBPs and men must agree to use adequate contraception (hormonal or
barrier method of birth control; abstinence) prior to study entry, for the duration of
study participation, and for 5 months after the last dose of pembrolizumab and 28 days
after the last dose of ipatasertib. Should a woman become pregnant or suspect she is
pregnant while she or her partner is participating in this study, she should inform
her treating physician immediately.
• Patients with grade >= 2 uncontrolled or untreated hypercholesterolemia or
hypertriglyceridemia are excluded.
• Patient has a known additional malignancy that is progressing or requires active
treatment. Exceptions include basal cell carcinoma of the skin, squamous cell
carcinoma of the skin, or carcinoma in situ (e.g., breast carcinoma, cervical cancer)
that has undergone potentially curative therapy.
• History of or active inflammatory bowel disease (e.g., Crohn's disease and ulcerative
colitis) or active bowel inflammation (e.g., diverticulitis).
• Lung disease: pneumonitis, interstitial lung disease, idiopathic pulmonary fibrosis,
cystic fibrosis, aspergillosis, active tuberculosis, or history of opportunistic
infections (pneumocystis pneumonia or cytomegalovirus pneumonia).
• Known clinically significant history of liver disease consistent with Child Pugh Class
B or C, including active viral or other hepatitis (e.g., positive for hepatitis B
surface antigen [HbsAg] or hepatitis C virus [HCV] antibody at screening), current
drug or alcohol abuse, or cirrhosis.
Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Squamous Cell Carcinoma, Metastatic Hypopharyngeal Squamous Cell Carcinoma, Metastatic Laryngeal Squamous Cell Carcinoma, Metastatic Oral Cavity Squamous Cell Carcinoma, Metastatic Oropharyngeal Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma, Recurrent Hypopharyngeal Squamous Cell Carcinoma, Recurrent Laryngeal Squamous Cell Carcinoma, Recurrent Oral Cavity Squamous Cell Carcinoma, Recurrent Oropharyngeal Squamous Cell Carcinoma, Stage IV Hypopharyngeal Carcinoma AJCC v8, Stage IV Laryngeal Cancer AJCC v8, Stage IV Lip and Oral Cavity Cancer AJCC v8, Stage IV Oropharyngeal (p16-Negative) Carcinoma AJCC v8, Lip, Oral Cavity and Pharynx, Head and Neck
Intervention to Improve the Delivery of Follow-up Care for Low-Risk Breast Cancer
This study tests a novel intervention designed to optimize needed survivorship care for
low-risk breast cancer survivors while reducing burdensome care with limited health benefits.
This study examines whether the intervention, titled REASSURE, improves survivors'
preparedness for survivorship. Up to 110 participants will be on study for up to 18 months.
Heather Neuman, MD
Female
18 Years to 95 Years old
N/A
This study is NOT accepting healthy volunteers
NCT05609435
Show full eligibility criteria
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Inclusion Criteria:
• Diagnosis of stage I, ER and/or PR positive, her2neu negative breast cancer within the
last 6-24 monoths
• Willing to complete study procedures using email
Exclusion Criteria:
• Receipt of chemotherapy for the stage I ER and/or PR positive, her2neu negative breast
cancer diagnosed within the last 6-24 months
• Participants will be excluded if they are unable to read and write in English or if,
in the opinion of a treating clinician, have cognitive impairment that would prevent
completion of study procedures
• Pregnancy, based on patient self-report. If a patient becomes pregnant during the
study period, they will be removed from the study at that time.
• Diagnosis of a ER and PR negative, her2neu negative breast cancer or a her2neu
positive breast cancer within the last 5 years
A Study to Compare the Administration of Encorafenib + Binimetinib + Nivolumab Versus Ipilimumab + Nivolumab in BRAF-V600 Mutant Melanoma With Brain Metastases
This phase II trial compares the effect of encorafenib, binimetinib, and nivolumab versus
ipilimumab and nivolumab in treating patients with BRAF- V600 mutant melanoma that has spread
to the brain (brain metastases). Encorafenib and binimetinib may stop the growth of tumor
cells by blocking some of the enzymes needed for cell growth. Ipilimumab and nivolumab are
monoclonal antibodies that may interfere with the ability of tumor cells to grow and spread.
This trial aims to find out which approach is more effective in shrinking and controlling
brain metastases from melanoma.
Vincent Ma, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04511013
Show full eligibility criteria
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Inclusion Criteria:
• Participants must have histologically and pathologically confirmed melanoma that has
metastasized to the brain
• Any primary (cutaneous, acral/mucosal, etc) or unknown origin are permitted, except
that participants with uveal primary are not eligible
• Participants must have BRAF-V600 mutant melanoma documented by a Clinical Laboratory
Improvement Act (CLIA)-certified laboratory
• All participants must have an magnetic resonance imaging (MRI) of the brain within 28
days prior to registration and must have central nervous system metastases with at
least one measurable brain metastasis >= 0.5 cm in size (per modified RECIST 1.1) that
has not been irradiated, or progressed (in the opinion of the treating physician)
after prior radiation therapy. Participating sites MUST use MRI slice thickness of =<
1.5 mm and are recommended to adhere to the 'minimum' Brain Tumor Imaging Protocol for
Clinical Trials in Brain Metastases (BTIP-BM) compliant MRI acquisition protocol.
Computed tomography (CT) of the head cannot substitute for brain MRI. (NOTE: All
central nervous system [CNS] disease must be documented on BOTH the Brain Metastases
Baseline Tumor Assessment Form, using modified RECIST, and the Baseline Tumor
Assessment Form [RECIST 1.1] using RECIST 1.1.)
• Participants may have measurable or non-measurable extracranial disease. All
measurable disease must be assessed within 28 days prior to randomization; all
non-measurable disease must be assessed within 42 days prior to randomization. Please
note, while any extracranial disease will also be assessed and followed, participants
are NOT required to have extracranial disease for randomization. NOTE: All disease
must be assessed and documented on the Baseline Tumor Assessment Form (RECIST 1.1).
CNS disease must be documented on BOTH the Brain Metastases Baseline Tumor Assessment
Form, using modified RECIST, and the Baseline Tumor Assessment Form (RECIST 1.1) using
RECIST 1.1
• Participants may have leptomeningeal disease
• Participants may be receiving corticosteroids for brain metastases at a dose of up to
8 mg of dexamethasone per day. The dose must not have exceeded 8 mg per day for at
least 7 days prior to randomization
• Participants must have Zubrod performance status =< 2
• Participants must have complete history and physical examination within 28 days prior
to randomization
• Participants must be able to swallow and retain pills
• Hemoglobin >= 8.0 g/dL (within 28 days prior to randomization)
• Absolute neutrophil count >= 1,500/mcL (within 28 days prior to randomization)
• Platelets >= 75,000/mcL (within 28 days prior to randomization)
• Total bilirubin =< 1.5 institutional upper limit of normal (ULN) (within 28 days prior
to randomization)
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.5 x
institutional ULN (in participants with liver metastases =< 5 x ULN) (within 28 days
prior to randomization)
• Creatinine =< 2.0 institutional ULN (within 28 days prior to randomization)
• Participants with known history or current symptoms of cardiac disease, or history of
treatment with cardiotoxic agents, must have a clinical risk assessment of cardiac
function using the New York Heart Association Functional Classification. To be
eligible for this trial, participants must be class 2B or better
• Participants with a prior or concurrent malignancy whose natural history or treatment
(in the opinion of the treating physician) does not have the potential to interfere
with the safety or efficacy assessment of the investigational regimen are eligible for
this trial
• Participants with known human immunodeficiency virus (HIV)-infection are eligible
providing they are on effective anti-retroviral therapy and have undetectable viral
load at their most recent viral load test and within 90 days prior to randomization
• Participants with a known history of hepatitis C virus (HCV) infection must have been
treated and cured. Participants with HCV infection who are currently on treatment must
have an undetectable HCV viral load prior to randomization
• Participants must agree to participate in image banking. Images must be submitted via
the Triad System
• Participants must be offered the opportunity to participate in specimen and blood
collections
• Participants must be informed of the investigational nature of this study and must
sign and give informed consent in accordance with institutional and federal guidelines
• As a part of the OPEN registration process the treating institution's identity is
provided in order to ensure that the current (within 365 days) date of institutional
review board approval for this study has been entered in the system
Exclusion Criteria:
• Participants must not have received prior systemic therapy for metastatic disease.
Prior systemic therapy received only in the neoadjuvant and/or adjuvant setting (e.g.,
BRAF/MEK inhibitor therapy, anti-PD-1 therapy or anti-CTLA4 therapy, alfa-interferon,
etc.) is permitted. If patients received prior neoadjuvant/adjuvant therapy, they must
have had eventual disease relapse prior to randomization
• Participants must not have had prior radiation therapy within 7 days prior to
randomization
• Participants must not be planning to require any additional form of systemic
anti-tumor therapy for melanoma while on protocol treatment
• Participants must not be planning to use hormonal contraceptives
• Participants must not have a serious active infection requiring systemic therapy at
time of randomization in the opinion of the treating physician
• Participants must not have active autoimmune disease that has required treatment in
the past 6 months with use of biologic disease modifying agents (.e.g. infliximab,
adalimumab). Patients on non-biologic disease modifying agents (e.g. methotrexate) or
patients on corticosteroids =< 10 mg prednisone daily or equivalent (to treat
auto-immune disease), or on replacement therapy (e.g., thyroxine, insulin) are
eligible if deemed in the best interest of the patient by treating physician
• Participants must not have had grade 3 or 4 immune-related adverse events on
ipilimumab or nivolumab that required more than 12 weeks of immune suppression with
corticosteroids
• Participants must not have had adverse events related to encorafenib and/or
binimetinib specifically, that required discontinuation of one or both drugs. (Please
note this does not apply to other BRAF/MEK inhibitor drugs.)
• Participants must not be pregnant or nursing. Women/men of reproductive potential must
have agreed to use an effective method of contraception. (NOTE: Patients must agree to
not use hormonal contraceptives, as encorafenib can result in decreased concentration
and loss of efficacy.) A woman is considered to be of "reproductive potential" if she
has had menses at any time in the preceding 12 consecutive months. In addition to
routine contraceptive methods, "effective contraception" also includes heterosexual
celibacy and surgery intended to prevent pregnancy (or with a side-effect of pregnancy
prevention) defined as a hysterectomy, bilateral oophorectomy or bilateral tubal
ligation. However, if at any point a previously celibate participant chooses to become
heterosexually active during the time period for use of contraceptive measures
outlined in the protocol, he/she is responsible for beginning contraceptive measures
Acral Lentiginous Melanoma, Clinical Stage IV Cutaneous Melanoma AJCC v8, Metastatic Cutaneous Melanoma, Metastatic Malignant Neoplasm in the Brain, Metastatic Melanoma, Metastatic Mucosal Melanoma, Pathologic Stage IV Cutaneous Melanoma AJCC v8, Melanoma, Skin, Melanoma/Skin cancer
GD2-SADA:177Lu-DOTA Complex in Patients With Solid Tumors Known to Express GD2
Patients with Small Cell Lung Cancer, Sarcoma and Malignant Melanoma will be treated with
GD2-SADA:177Lu-DOTA complex(The IMP is a two-step radioimmunotherapy, delivered as two
separate products GD2-SADA and 177Lu-DOTA) to assess safety and tolerability
Vincent Ma, MD
All
16 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT05130255
Show full eligibility criteria
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Inclusion Criteria:
• Signed informed consent from patient, legal guardian(s) and/or adolescents obtained in
accordance with local regulations. Pediatric patients must provide assent as required
by local regulations.
• Age ≥18 years at the time of informed consent, for sarcoma age ≥16 years of age at
time of informed consent/assent
• Measurable disease according to RECIST 1.1
• ECOG performance status 0-1
• Expected survival >3 months
• Platelet counts ≥100,000 cells/mm3
• Hemoglobin ≥9 g/dL
• Adequate renal function with serum creatinine ≤1.5 mg/dL or creatinine clearance
≥60mL/min as calculated using the Cockcroft-Gault equation
• Patient willing and able to comply with the trial protocol
Exclusion Criteria:
• Systemic chemotherapy, radiotherapy, immunotherapy, or major surgery administered
within 3 weeks prior to the first planned dosing of the IMP per protocol
• Patients receiving any other investigational therapy for their cancer within 3 weeks
prior to the first planned dosing of the IMP per protocol
• Ongoing radiation toxicities from prior RT therapy
• Patients with a diagnosis of autoimmune diseases or immunodeficiencies or documented
infection with human immunodeficiency virus (HIV) or hepatitis B or C virus (active)
• Prior treatment with anti-GD2 antibody
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the
treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine
receptor (AChR).
Stephanie Gardon
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05556096
Show full eligibility criteria
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Inclusion Criteria:
• Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined
by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
• Positive serological test for autoantibodies against AChR
Exclusion Criteria:
• History of thymectomy, or any other thymic surgery within 12 months prior to Screening
• Untreated thymic malignancy, carcinoma, or thymoma
• History of Neisseria meningitidis infection
• Pregnancy, breastfeeding, or intention to conceive during the course of the study
Generalized Myasthenia Gravis, Myasthenia gravis and other myoneural disorders, Other, Brain & Neurological
Chemotherapy for the Treatment of Patients With Newly Diagnosed Very Low-Risk and Low Risk Fusion Negative Rhabdomyosarcoma
Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase
III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma
(VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine
and dactinomycin (VA) and examines the use of centralized molecular risk stratification in
the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients
with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with
VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment.
Finally, this study examines the effect of therapy intensification in patients who have RMS
cancer with DNA mutations to see if their outcomes can be improved.
Kenneth Desantes, M.D.
All
up to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05304585
Show full eligibility criteria
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Inclusion Criteria:
• All patients must be enrolled on APEC14B1 (NCT02402244) and consented to the Molecular
Characterization Initiative (Part A) prior to enrollment and treatment on ARST2032
(this trial).
• Patients must be =< 21 years at the time of enrollment.
• Patients must have newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle
cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma (ARMS)
(institutional FOXO1 fusion results are acceptable). RMS types included under ERMS
include those classified in the 1995 International Classification of Rhabdomyosarcoma
(ICR) as ERMS (classic, spindle cell, and botryoid variants), which are reclassified
in the 2020 World Health Organization (WHO) classification as ERMS (classic, dense and
botryoid variants) and spindle cell/sclerosing RMS (encompassing the historical
spindle cell ERMS variant and the newly recognized sclerosing RMS variant). Enrollment
in APEC14B1 is required for all patients.
• All patients will be evaluated for stage and clinical group. Note that clinical
group designation assigned at the time of enrollment on study remains unchanged
regardless of any second-look operation that may be performed.
• Patients will be eligible for the very low-risk stratum (Regimen VA) if they
have Stage 1, CG I disease.
• Patients will be eligible for the low-risk stratum (Regimen VAC/VA) if they
have Stage 1, CG II disease, Stage 2, CG I or II disease, or Stage 1, CG III
(orbit only) disease.
• Paratesticular Tumors: Staging ipsilateral retroperitoneal lymph node sampling
(SIRLNS) is required for all patients >= 10 years of age with paratesticular
tumors who do not have gross nodal involvement on imaging.
• Extremity Tumors: Regional lymph node sampling is required for histologic
evaluation in patients with extremity tumors.
• Clinically or radiographically enlarged nodes must be sampled for histologic
evaluation.
• Patients must have a Lansky (for patients =< 16 years of age) or Karnofsky (for
patients > 16 years of age) performance status score of >= 50. Patients who are unable
to walk because of paralysis, but who are up in a wheelchair, will be considered
ambulatory for the purpose of assessing performance score.
• Peripheral absolute neutrophil count (ANC) >= 750/uL (within 7 days prior to
enrollment).
• Platelet count >= 75,000/uL (transfusion independent) (within 7 days prior to
enrollment).
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine (within 7 days prior to enrollment) based on
age/gender as follows:
• Age: 1 month to < 6 months; Maximum serum creatinine (mg/dL): 0.4 (male) : 0.4
(female)
• Age: 6 months to < 1 year; Maximum serum creatinine (mg/dL): 0.5 (male) : 0.5
(female)
• Age: 1 to < 2 years; Maximum serum creatinine (mg/dL): 0.6 (male) : 0.6 (female)
• Age: 2 to < 6 years; Maximum serum creatinine (mg/dL): 0.8 (male) : 0.8 (female)
• Age: 6 to < 10 years; Maximum serum creatinine (mg/dL): 1 (male) : 1 (female)
• Age: 10 to < 13 years; Maximum serum creatinine (mg/dL): 1.2 (male) : 1.2
(female)
• Age: 13 to < 16 years; Maximum serum creatinine (mg/dL): 1.5 (male) : 1.4
(female)
• Age >= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male) : 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment), and
• If there is evidence of biliary obstruction by the tumor, then the total
bilirubin must be < 3 x ULN for age.
• Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the
value of 45 U/L.
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L (within 7 days prior to enrollment).
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Exclusion Criteria:
• Patients who have received prior chemotherapy and/or radiation therapy for cancer
prior to enrollment. Surgical resection alone of previous cancer(s) is permitted.
• Patients who have received chemotherapy or radiation for non-malignant conditions
(e.g., autoimmune diseases) are eligible. Patients must discontinue chemotherapy for
non-malignant conditions prior to starting protocol therapy.
• Vincristine is sensitive substrate of the CYP450 3A4 isozyme. Patients must not have
received drugs that are moderate to strong CYP3A4 inhibitors and inducers within 7
days prior to study enrollment.
• Patients unable to undergo radiation therapy, if necessary, as specified in the
protocol.
• Evidence of uncontrolled infection.
• Female patients who are pregnant since fetal toxicities and teratogenic effects have
been noted for several of the study drugs. A pregnancy test is required for female
patients of childbearing potential.
• Lactating females who plan to breastfeed their infants.
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation.
Embryonal Rhabdomyosarcoma, Fusion-Negative Alveolar Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma, Lip, Oral Cavity and Pharynx, Esophagus, Stomach, Small Intestine, Colon, Rectum, Liver, Pancreas, Other Digestive Organ, Larynx, Lung, Other Respiratory and Intrathoracic Organs, Soft Tissue, Ovary, Other Female Genital, Prostate, Other Male Genital, Urinary Bladder, Kidney, Other Urinary, Eye and Orbit, Thyroid, Other Endocrine System, Bladder, Colon and Rectum, Endocrine cancers, Gastrointestinal cancers, other, Genitourinary cancers, other, Head and Neck, Melanoma/Skin cancer, Sarcoma
A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCT
This phase II trial studies the best approach to combine chemotherapy and radiation therapy
(RT) based on the patient's response to induction chemotherapy in patients with
non-germinomatous germ cell tumors (NGGCT) that have not spread to other parts of the brain
or body (localized). This study has 2 goals: 1) optimizing radiation for patients who respond
well to induction chemotherapy to diminish spinal cord relapses, 2) utilizing higher dose
chemotherapy followed by conventional RT in patients who did not respond to induction
chemotherapy. Chemotherapy drugs, such as carboplatin, etoposide, ifosfamide, and thiotepa,
work in different ways to stop the growth of tumor cells, either by killing the cells, by
stopping them from dividing, or by stopping them from spreading. Radiation therapy uses high
energy x-rays or high-energy protons to kill tumor cells and shrink tumors. Studies have
shown that patients with newly-diagnosed localized NGGCT, whose disease responds well to
chemotherapy before receiving radiation therapy, are more likely to be free of the disease
for a longer time than are patients for whom the chemotherapy does not efficiently eliminate
or reduce the size of the tumor. The purpose of this study is to see how well the tumors
respond to induction chemotherapy to decide what treatment to give next. Some patients will
be given RT to the spine and a portion of the brain. Others will be given high dose
chemotherapy and a stem cell transplant before RT to the whole brain and spine. Giving
treatment based on the response to induction chemotherapy may lower the side effects of
radiation in some patients and adjust the therapy to a more efficient one for other patients
with localized NGGCT.
Kenneth Desantes, M.D.
All
3 Years to 29 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04684368
Show full eligibility criteria
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Inclusion Criteria:
• Patients must be >= 3 years and < 30 years at the time of study enrollment
• Patients must be newly diagnosed with localized primary CNS NGGCT of the suprasellar
and/or pineal region by pathology and/or serum or cerebrospinal fluid (CSF) elevation
of AFP above institutional normal or > 10 ng/mL or human chorionic gonadotropin (hCG)
beta > 100 mIU/mL as confirmed by Rapid Central Marker Screening Review on
APEC14B1-CNS. Suprasellar, pineal and bifocal tumors are included. (CSF tumor markers
and cytology must be within 31 days prior to enrollment and start of protocol therapy
[repeat if necessary]. Serum tumor markers, AFP and hCGbeta must be within 7 days
prior to enrollment and start of protocol therapy [repeat if necessary]). Basal
ganglia or other primary sites are excluded
• Patients with any of the following pathological elements are eligible: endodermal
sinus (yolk sac), embryonal carcinoma, choriocarcinoma, malignant/immature teratoma
and mixed germ cell tumor (GCT) (i.e., may include some pure germinoma) if malignant
elements listed above are present. Patients with only mature teratoma are excluded.
Patients with pure germinoma admixed with mature teratoma are excluded (would be
eligible for pure germinoma protocols)
• Patients must have a cranial MRI with and without gadolinium at diagnosis/prior to
enrollment. If surgical resection is performed, patients must have pre-operative and
post operative brain MRI with and without gadolinium. The post operative brain MRI
should be obtained within 72 hours of surgery. If patient has a biopsy only,
post-operative brain MRI is recommended but not required (within 31 days prior to
study enrollment and start of protocol therapy )
• Patients must have a spine MRI with gadolinium obtained at diagnosis/prior to
enrollment. Spine MRI with and without gadolinium is recommended (within 31 days prior
to study enrollment and start of protocol therapy)
• Lumbar CSF must be obtained prior to study enrollment unless medically
contraindicated. If a patient undergoes surgery and lumbar CSF cytology cannot be
obtained at the time of surgery, then it should be performed at least 10 days
following surgery and prior to study enrollment. False positive cytology can occur
within 10 days of surgery
• Patients must have RAPID CENTRAL TUMOR MARKER REVIEW CSF tumor markers obtained prior
to enrollment unless medically contraindicated. Ventricular CSF obtained at the time
of CSF diversion procedure (if performed) is acceptable for tumor markers but lumbar
CSF is preferred. In case CSF diversion and biopsy/surgery are combined, CSF tumor
markers should be collected first
• Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to
enrollment)
• Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to
enrollment)
• Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (within 7 days
prior to enrollment)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows (within 7 days
prior to enrollment):
• Age: Maximum serum creatinine (mg/dL)
• 3 to < 6 years: 0.8 (male), 0.8 (female)
• 6 to < 10 years: 1 (male), 1 (female)
• 10 to < 13 years: 1.2 (male), 1.2 (female)
• 13 to < 16 years: 1.5 (male), 1.4 (female)
• >= 16 years: male (1.7), 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment)
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L (within 7 days prior to enrollment)
• Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the
value of 45 U/L
• Central nervous system function defined as:
• Patients with seizure disorder may be enrolled if on anticonvulsants and well
controlled
• Patients must not be in status epilepticus, coma or assisted ventilation prior to
study enrollment
• Protocol therapy must begin within 31 calendar days of definitive surgery or clinical
diagnosis, whichever is later. If a biopsy only was performed, the biopsy date will be
considered the date of definitive surgery. For patients who have a biopsy or
incomplete resection at diagnosis followed by additional surgery, the date of the last
resection will be considered the date of definitive surgery.
• All patients and/or their parents or legal guardians must sign a written informed
consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
• NEUROCOGNITIVE FUNCTION AND QUALITY OF LIFE ASSESSMENT:
• English-, Spanish-, or French- speaking
• Note: Patients who speak a language other than English, Spanish, or French will
be allowed to participate in ACNS2021 but will not complete the neurocognitive
and quality of life assessments
• No known history of neurodevelopmental disorder prior to diagnosis of NGGCT (e.g.,
Down syndrome, fragile X, William syndrome, intellectual disability). Patients with
NF1 will be allowed to participate
• Additional eligibility criteria for the COG Standardized Neuropsychological Battery
only: must be at a site that has a psychologist to administer the battery
• Note: If not eligible for the COG Standardized Battery, patients should still
complete the Behavior Rating Inventory of Executive Function, Second Edition
(BRIEF-2), Pediatric Quality of Life Inventory (PedsQL), Adaptive Behavior
Assessment System Third Edition (ABAS-3), and Behavior Assessment System for
Children, Third Edition (BASC-3) questionnaires
Exclusion Criteria:
• Patients with tumors located outside the ventricles (i.e., basal ganglia, thalamus)
• Patients with only mature teratoma and non-elevated markers upon tumor sampling at
diagnosis
• Patients who have received any prior tumor-directed therapy for their diagnosis of
NGGCT other than surgical intervention and corticosteroids
• Patients with metastatic disease (i.e., MRI evaluation, lumbar CSF cytology or
intraoperative evidence of dissemination)
• Female patients who are pregnant, since fetal toxicities and teratogenic effects have
been noted for several of the study drugs
• Note: Serum and urine pregnancy tests may be falsely positive due to
HCGbeta-secreting germ cell tumors. Ensure the patient is not pregnant by
institutional standards
• Lactating females who plan to breastfeed their infants
• Sexually active patients of reproductive potential who have not agreed to use an
effective contraceptive method for the duration of their study participation
Central Nervous System Nongerminomatous Germ Cell Tumor, Choriocarcinoma, Embryonal Carcinoma, Immature Teratoma, Malignant Teratoma, Mixed Germ Cell Tumor, Pineal Region Germ Cell Tumor, Pineal Region Immature Teratoma, Pineal Region Yolk Sac Tumor, Suprasellar Germ Cell Tumor, Brain and Nervous System, Brain/Central Nervous System
This study will use different types of medical imaging to assess how lesions from advanced
prostate cancer become resistant to second-generation AR-targeted therapy, and how the
different types of imaging compare in that assessment. Participants in this study have
advanced prostate cancer and are either scheduled to start a second-generation androgen
receptor (AR) targeted therapy (such as enzalutamide, abiraterone, or apalutamide) or are
already being treated with one. Participants can expect to be in the study for at least 9
months, and up to 2 years.
Christos Kyriakopoulos, MD
Male
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT05647564
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Inclusion Criteria:
• Histologically proven adenocarcinoma of the prostate.
• At least 1 radiographic metastases as seen on conventional CT imaging or bone scan
• Progressive prostate cancer as evident by at least two separate increase in PSA over
nadir, and absolute PSA value at least 2 ng/ml (INTRINSIC RESISTANCE COHORT ONLY)
• Patients must be candidate for a second-generation androgen receptor (AR) inhibitor
(e.g. enzalutamide, abiraterone, apalutamide), or Lu177-PSMA radioligand therapy
(INTRINSIC RESISTANCE COHORT ONLY)
• Men of age >18 years.
• Patients must be able to comply with all study procedures, including having both the
ability and willingness to lie flat for ≥ 30 minutes during imaging
• Patients must be informed of the exploratory nature of the study and its potential
risks, and must sign IRB- approved consent form indicating such understanding.
• Life-expectancy at least 12 months
• Patients currently receiving a second-generation androgen receptor (AR) inhibitor
(e.g. enzalutamide, abiraterone, apalutamide) and must have had 1) PSA decline on
treatment and 2) now have PSA increase over nadir while still on treatment (patients
must be registered within 12 weeks of first documented PSA increase) (ACQUIRED
RESISTANCE COHORT ONLY)
Exclusion Criteria:
• Must not have uncontrolled diabetes (fasting blood sugar > 200 mg/dL or inability to
safely hold diabetes medication or fast 6 hours prior to FDG PET scan)
• Prior treatment with second-generation AR inhibitor for prostate cancer in the
metastatic disease setting (prior second-generation AR inhibitor in the neoadjuvant or
adjuvant setting is permitted unless patient developed progression while on treatment)
(INTRINSIC RESISTANCE COHORT, AR-INHIBITOR GROUP ONLY)
• Pain or clinical symptoms from metastatic prostate cancer requiring opioid analgesics
• Known neuro-endocrine prostate cancer
• Prior radioisotope therapy for castration-resistant prostate cancer
• To avoid the possibility of unintended coercion, vulnerable populations such as
incarcerated subjects, subjects unable to provide their own informed consent and
non-English speaking patients will not be considered
A Study of Zilovertamab Vedotin (MK-2140) as Monotherapy and in Combination in Participants With Aggressive and Indolent B-cell Malignancies (MK-2140-006)
The purpose of this study is to assess the safety and tolerability of zilovertamab vedotin as
monotherapy and in combination in participants with select B-cell lymphomas including mantle
cell lymphoma (MCL), Richter's transformation lymphoma (RTL), follicular lymphoma (FL), and
chronic lymphocytic leukemia (CLL). This study will also evaluate zilovertamab vedotin as
monotherapy and in combination with respect to objective response rate.
- Cohort A: Participants with relapsed or refractory MCL relapsed or refractory disease
after at least 2 prior systemic therapies including a Bruton's tyrosine kinase
inhibition/inhibitor (BTKi), and post therapy chimeric antigen receptor T (CAR-T) cell
therapy or ineligible for CAR-T cell therapy
- Cohort B: Participants with relapsed or refractory RT disease after at least 1 prior
systemic therapy
- Cohort C: Participants with relapsed or refractory MCL relapsed or refractory disease
after at least 1 prior systemic therapy and no prior exposure to a non-covalent BTKi
- Cohort D: Participants with relapsed or refractory FL and CLL relapsed or refractory
disease after at least 2 prior systemic therapies and have no other available therapy
- Cohort E: Participants with relapsed or refractory FL after at least 2 prior systemic
therapies and have no other available therapy
- Cohort F: Participants with relapsed or refractory CLL after at least 2 prior systemic
therapies and have no other available therapy
The primary study hypothesis is that zilovertamab vedotin monotherapy has an increased
Objective Response Rate (ORR) per Lugano Response Criteria as assessed by blinded independent
central review (BICR).
Christopher Fletcher, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05458297
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The main inclusion criteria include, but are not limited to the following:
Inclusion Criteria:
• For aggressive B-cell malignancies MCL: Has histologically confirmed biopsy according
to the 2016 World Health Organization (WHO) classification of neoplasms of the
hematopoietic and lymphoid tissues and has relapsed or refractory disease after at
least 2 prior systemic therapies including a Bruton's tyrosine kinase
inhibition/inhibitor(s) (BTKi), and is post chimeric antigen receptor T (CAR-T) cell
therapy or is ineligible for CAR-T cell therapy.
• For aggressive B-cell malignancies MCL Cohort C: Has histologically confirmed biopsy
according to the 2016 World Health Organization (WHO) classification of neoplasms of
the hematopoietic and lymphoid tissues and has relapsed or refractory disease after at
least 1 prior systemic therapy and has no prior exposure to a non-covalent BTKi.
• For aggressive B-cell malignancies Richter transformation lymphoma (RTL): Has
histologically confirmed biopsy according to the 2016 World Health Organization (WHO)
classification of neoplasms of the hematopoietic and lymphoid tissues and has relapsed
or refractory disease.
• For indolent B-cell malignancies FL and CLL: Has histologically confirmed biopsy and
has relapsed or refractory disease after at least 2 prior systemic therapies and no
other available therapy.
• Participants who are hepatitis B surface antigen (HBsAg) positive are eligible if they
have received hepatitis B virus (HBV) antiviral therapy for at least 4 weeks and have
undetectable HBV viral load prior to randomization/allocation.
• Have an ECOG performance status of 0 to 2 assessed within 7 days before cycle 1 day 1.
Exclusion Criteria:
• Has received solid organ transplant at any time.
• Has clinically significant (ie, active) cardiovascular disease: cerebral vascular
accident/stroke (<6 months prior to enrollment), myocardial infarction (<6 months
prior to enrollment), unstable angina (<6 months prior to enrollment), congestive
heart failure (New York Heart Association Classification Class ≥II), or serious
cardiac arrhythmia requiring medication.
• Has pericardial effusion or clinically significant pleural effusion.
• Has ongoing Grade >1 peripheral neuropathy.
• Has a demyelinating form of Charcot-Marie-Tooth disease.
• Has a history of a second malignancy, unless potentially curative treatment has been
completed with no evidence of malignancy for 2 years.
• Participants with FL who have transformed to a more aggressive type of lymphoma.
• Has received prior systemic anticancer therapy within 5 half-lives or 4 weeks (if
prior therapy was a monoclonal antibodies) or 2 weeks (if prior therapy was small
molecules like kinase inhibitors) prior to the first dose of study intervention.
• Has received prior radiotherapy within 28 days of start of study intervention.
Participants must have recovered from all radiation-related toxicities.
• Has ongoing corticosteroid therapy exceeding 30 mg daily of prednisone equivalent.
• Has received a live or live-attenuated vaccine within 30 days before the first dose of
study intervention.
• Has known active central nervous system (CNS) lymphoma involvement or active CNS
involvement by lymphoma.
• Has an active infection requiring systemic therapy.
• Has a known history of human immunodeficiency virus (HIV) infection.
• Active HBV or hepatitis C virus (HCV) infection.
• For Cohort C only: has any clinically significant gastrointestinal abnormalities that
might alter absorption.
MC1R-targeted Alpha-particle Therapy Trial in Adults With Advanced Melanoma
In this first-in human, phase I/IIa study, the safety and efficacy of [212Pb]VMT01, an
alpha-particle emitting therapeutic agent targeted to melanocortin sub-type 1 receptor (MC1R)
is being evaluated in patients with unresectable and metastatic melanoma.
Zachary Morris, MD
All
18 Years to 90 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT05655312
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Inclusion Criteria:
• Ability to understand and willingness to provide informed consent, willingness to
comply with all study procedures for the duration of the study
• Male or female, aged ≥ 18 years
• Diagnosed with Stage IV metastatic melanoma, or unresectable Stage III
• Previously progressed (clinical or radiological progression) on at least one prior
therapy for metastatic melanoma
• Uptake of [68Ga]VMT02 or [203Pb]VMT01 by PET or SPECT imaging observed in at least one
melanoma tumor site using quantitative imaging analysis compared to reference normal
tissue
• Subjects on prior intravenous therapy (e.g., chemotherapy or checkpoint inhibitors),
or prior oral therapy (e.g., BRAF or MEK inhibitors) who demonstrate MC1R positivity
during screening are eligible for enrollment, provided that they undergo a wash-out
period of 21 days, or 14 days, respectively, prior to Day 1 treatment with
[212Pb]VMT01.
• Presence of measurable disease by RECIST v1.1 criteria assessed within 30 days prior
to the start of Day 1
• Ability to lie flat and still for up to two hours for imaging scans; moderate
conscious sedation allowed if indicated
• For females of reproductive potential: use of highly effective contraception for at
least one month prior to screening, and agreement to use such a method during study
participation and for an additional four weeks after the last administration of an
investigational product
• For males of reproductive potential: use of condoms or other methods to ensure
effective contraception with partner during study participation and for an additional
four weeks after the last administration of an investigational product
• ECOG performance score of < 2 at Screening
• Life expectancy of at least 3 months
• Evidence of sufficient organ function as determined by all of the following:
Oxygen saturation > 90% on room air eGFR > 50 mL/min/1.73m2 by CKD-EPI equation Complete
blood count with differential, within 7 calendar days prior to therapy and off Growth
Factors White blood cells (WBC) > 2500/mm3 Hemoglobin (Hgb) > 9.0 g/dL Platelets >
60,000/mm3 Absolute Neutrophil Count (ANC) > 1,250/mm3
The comprehensive metabolic panel, within seven calendar days prior to Day 1, demonstrating
values within the site's upper limit of normal (ULN), with the following exceptions:
Alanine aminotransferase (ALT) < 3x ULN Aspartate aminotransferase (AST) < 3x ULN Alkaline
phosphatase (ALP) < 2.5x ULN
Exclusion Criteria:
• Active secondary malignancy
• Prior treatment (for any reason) with radioactive nuclides; however, imaging tracers
are acceptable
• Pregnancy or breastfeeding a child
• Active infection
• Brain metastasis requiring acute therapy of any modality (i.e., surgical or external
beam radiotherapy) within two weeks of enrollment or clinical instability, including
signs or symptoms of brain edema. Subjects must demonstrate stable or decreasing brain
metastasis by a noninvasive imaging scan and must be off steroids or on decreasing
doses prior to enrollment.
• Treatment with another investigational drug product (therapeutic IND agents) within
the last 30 days.
• Current abuse of alcohol or illicit drugs
• Existence of any medical or social issues likely to interfere with study conductor
that may cause increased risk to the subject or to others, e.g., lack of ability to
follow radiation safety precautions
Melanoma, Skin, Melanoma/Skin cancer, Melanoma (Skin), Metastatic Melanoma, Melanoma Stage IV, Melanoma, Uveal, Mucosal Melanoma, Melanoma Stage III
An Observational Study of the Progression of Intermediate Age-Related Macular Degeneration (HONU)
This is a multicenter prospective study in participants with intermediate age-related macular
degeneration (iAMD). One primary objective of this study is to assess iAMD disease
progression, by the timeline and rates of conversion for high-risk iAMD at baseline to more
advanced atrophic AMD stages. The other primary objective of this observational study is to
assess the feasibility of measuring the rate of photoreceptor loss as a potential clinical
endpoint. The study will consist of an observation period of approximately 3 years (~144
weeks) for participants.
Mihai Mititelu, MD, MPH
All
50 Years to 94 Years old
N/A
This study is NOT accepting healthy volunteers
NCT05300724
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Inclusion Criteria:
• For women of childbearing potential, agreement to remain abstinent (refrain from
heterosexual intercourse) or use contraception, during the study for at least 28 days
after the last fluorescein injection for the fluorescein angiography (FA)
administration
• Study eye: High-risk intermediate AMD
Exclusion Criteria:
• Macular disease in either eye with subretinal deposits not typical of AMD
• Pigmentary abnormalities of the retina in either eye not typical of AMD
• Atrophy in either eye due to causes other than AMD
• Study eye: Any concurrent or history of ocular or intraocular condition
• Study eye: Intraocular surgery, including cataract surgery, within 3 months prior to
Day 1
• Study eye: Retinal tears or peripheral retinal breaks within 3 months prior to Day 1
• Study eye: Concurrent or history of retinal laser photocoagulation or anti-vascular
endothelial growth factor (anti-VEGF) treatment for exudative MNV, diabetic macular
edema, retinal vein occlusion, or proliferative diabetic retinopathy
• Study eye: Presence of choroidal nevus with overlying drusen in the circle with a
radius 3600 micrometer centered on the fovea
• Study eye: Previous participation in interventional clinical trials for GA or early
stages of AMD, except for vitamins and minerals, regardless of the route of
administration within the last 6 months, except for sham-arm participants
• Study eye: History of glaucoma surgery, corneal transplant, retinal pigment epithelium
tear, retinal tear that involves the macula, retinal detachment
• Either eye: Uncontrolled progressive glaucoma
• Either eye: Moderate or severe non-proliferative diabetic retinopathy or proliferative
diabetic retinopathy
• Either eye: History of recurrent infectious or inflammatory ocular disease
• Any concurrent or history of taking medications that can induce retinal toxicity
Testing the Addition of Trastuzumab or Trastuzumab/Pertuzumab to the Usual Chemotherapy for HER2 Positive Endometrial Serous Carcinoma or Carcinosarcoma
This phase II/III trial tests whether adding trastuzumab and hyaluronidase-oysk (Herceptin
HylectaTM) or pertuzumab, trastuzumab and hyaluronidase-zzxf (PhesgoTM) to the usual
chemotherapy (paclitaxel and carboplatin) works to shrink tumors in patients with HER2
positive endometrial serous carcinoma or carcinosarcoma. Trastuzumab and pertuzumab are
monoclonal antibodies and forms of targeted therapy that attach to specific molecules
(receptors) on the surface of tumor cells, known as HER2 receptors. When trastuzumab or
pertuzumab attach to HER2 receptors, the signals that tell the cells to grow are blocked and
the tumor cell may be marked for destruction by the body's immune system. Hyaluronidase is an
endoglycosidase. It helps to keep pertuzumab and trastuzumab in the body longer, so that
these medications will have a greater effect. Hyaluronidase also allows trastuzumab and
trastuzumab/pertuzumab to be given by injection under the skin and shortens their
administration time compared to trastuzumab or pertuzumab alone. Paclitaxel is a taxane and
in a class of medications called antimicrotubule agents. It stops cancer cells from growing
and dividing and may kill them. Carboplatin is in a class of medications known as
platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin,
but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing
the growth of tumor cells. Giving Herceptin Hylecta or Phesgo in combination with paclitaxel
and carboplatin may shrink the tumor and prevent the cancer from coming back in patients with
HER2 positive endometrial serous carcinoma or carcinosarcoma.
Ellen Hartenbach, M.D.
Female
18 Years and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT05256225
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Inclusion Criteria:
• Federation of Gynecology and Obstetrics (FIGO) 2009 stage IA-IVB, non-recurrent,
chemotherapy (chemo)-naive, HER2-positive endometrial serous carcinoma or endometrial
carcinosarcoma
• Histologic confirmation of the original primary tumor is required. Submission of
surgical pathology report (or endometrial biopsy pathology report in patients who
never undergo hysterectomy) is required
• Patients must be within 8 weeks of primary surgery (or endometrial biopsy in patients
who never undergo hysterectomy) at the time of study registration
• Patients may have measurable disease, non-measurable disease, or no measurable
disease. In patients with measurable disease, lesions will be defined and monitored by
RECIST v 1.1. Measurable disease is defined as at least one lesion that can be
accurately measured in at least one dimension (longest diameter to be recorded). Each
lesion must be >= 10 mm when measured by computed tomography (CT) or magnetic
resonance imaging (MRI). Lymph nodes must be >= 15 mm in short axis when measured by
CT or MRI
• For patients with uterine-confined (stage I) disease, the tumor must be invasive into
the myometrium. Any amount of myoinvasion is acceptable for eligibility. Patients with
non-invasive disease, endometrial intraepithelial carcinoma alone, or disease confined
to a polyp will be excluded
• Additionally, patients must have the following histologic types to be eligible:
• Serous adenocarcinoma (may include =< 10% non-serous histology)
• Carcinosarcoma with serous epithelial component (only the serous component needs
to be HER2 positive; may include =< 10% non-serous histology)
• In cases where determination of serous is equivocal or challenging, aberrant p53
immunohistochemistry (IHC) (defined as overexpression of p53 compared to internal
controls) will be sufficient for inclusion
• All patients must have tumors that are HER2 positive as defined by American Society of
Clinical Oncology (ASCO)/College of American Pathologists (CAP) 2018 Breast Cancer
guidelines (https://documents.cap.org/documents/algorithim-evaluation-her2.pdf. In
general HER2 positivity is defined as any of the following:
• 3+ immunohistochemistry (IHC),
• 2+ IHC with positive in situ hybridization (ISH)
• Average HER2 copy number >= 6.0 signals/cell
• Average HER2 copy number >= 4.0 and < 6.0 signals/cell, with concurrent IHC 3+
• HER2/CEP17 ratio >= 4.0 signals/cell
• HER2/CEP 17 ratio >= 2.0 and < 4.0, with concurrent IHC 3+ IHC and ISH testing
will be done locally, at each participating institution and interpreted by local
pathologists. Alternatively, patients could be eligible if next generation
sequencing (NGS) demonstrates HER2 (ERBB2) amplification. NGS testing can be
performed through any designated labs as per the National Cancer Institute (NCI)
MATCH/NCI Combo-MATCH trial
(https://ecog-acrin.org/nci-match-eay131-designated-labs).
Pathology report showing results of institutional HER2 testing (or NGS testing results)
must be submitted.
Sites must submit all results available (IHC, ISH, and NGS)
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
• Age >= 18
• Platelets >= 100,000/mcl (within 14 days prior to registration)
• Absolute neutrophil count (ANC) >= 1,500/mcl (within 14 days prior to registration)
• Creatinine =< 1.5 x institutional/laboratory upper limit of normal (ULN) or estimated
Glomerular filtration rate (eGFR) >= 50 mL/min using either the Cockcroft-Gault
equation, the Modification of Diet in Renal Disease Study, or as reported in the
comprehensive metabolic panel/basic metabolic panel (eGFR). (within 14 days prior to
registration)
• Total serum bilirubin level =< 1.5 x ULN (patients with known Gilbert's disease who
have bilirubin level =< 3 x ULN may be enrolled) (within 14 days prior to
registration)
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 3 x ULN (within
14 days prior to registration)
• Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral
therapy with undetectable viral load within 6 months of registration are eligible for
this trial
• Although the uterus will have been removed in the vast majority of patients, for
patients of child-bearing potential: negative urine or serum pregnancy test. If the
urine test is positive or cannot be confirmed as negative, a serum pregnancy test is
required. Patients will be considered of non-reproductive potential if they are
either:
• Postmenopausal (defined as at least 12 months with no menses without an
alternative medical cause; in women < 45 years of age, a high follicle
stimulating hormone [FSH] level in the postmenopausal range may be used to
confirm a postmenopausal state in women not using hormonal contraception or
hormonal replacement therapy. In the absence of 12 months of amenorrhea, a single
FSH measurement is insufficient); OR
• Have had a hysterectomy and/or bilateral oophorectomy, bilateral salpingectomy or
bilateral tubal ligation/occlusion at least 6 weeks prior to registration
• Have a congenital or acquired condition that prevents childbearing
• Patients with a prior or concurrent malignancy whose natural history or treatment does
not have the potential to interfere with the safety or efficacy assessment of the
investigational regimen are eligible for this trial. NOTE: Patients with prior
anthracycline exposure are NOT eligible
• Patients with evidence of chronic hepatitis B virus (HBV) infection must have an
undetectable HBV viral load on suppressive therapy, if indicated
• Patients with a history of hepatitis C virus (HCV) infection must have been treated
and cured. For patients with HCV infection who are currently on treatment, they are
eligible if they have an undetectable HCV viral load
• Patients with treated brain metastases are eligible if follow-up brain imaging after
central nervous system (CNS)-directed therapy shows no evidence of progression
• The patient or a legally authorized representative must provide study-specific
informed consent prior to study entry and, for patients treated in the United States
(U.S.), authorization permitting release of personal health information
Exclusion Criteria:
• Prior Therapy:
• Patients must NOT have received prior chemotherapy, biologic therapy, or targeted
therapy for treatment of endometrial carcinoma
• Patients must NOT have received prior radiation therapy for treatment of
endometrial carcinoma. Prior radiation includes external beam pelvic radiation
therapy, external beam extended field pelvic/para-aortic radiation therapy,
and/or intravaginal brachytherapy
• NOTE: Vaginal brachytherapy for treatment of endometrial cancer is permitted
during study treatment. Planned use of vaginal brachytherapy must be
declared at time of registration
• Patients may have received prior hormonal therapy for treatment of endometrial
carcinoma. All hormonal therapy must be discontinued at least one week prior to
registration
• Patients may not have a planned interval cytoreduction or hysterectomy, prior to
documentation of progression, after study registration
• Patients may not have planned external beam radiotherapy, prior to documentation of
progression, after study registration
• Significant cardiovascular disease including:
• Uncontrolled hypertension, defined as systolic > 150 mm Hg or diastolic > 90 mm
Hg despite antihypertensive medications
• Myocardial infarction or unstable angina within 6 months prior to registration
• New York Heart Association functional classification II, III or IV
• Serious cardiac arrhythmia requiring medication. This does not include
asymptomatic, atrial fibrillation with controlled ventricular rate
• Significant lung disease: dyspnea at rest grade 2 or greater (resulting from extensive
tumor involvement or other causes), pneumonitis grade 2 or greater, interstitial lung
disease grade 2 or greater, idiopathic pulmonary fibrosis, cystic fibrosis,
Aspergillosis, active tuberculosis, or history of opportunistic infections
(pneumocystis pneumonia or cytomegalovirus pneumonia)
• Patients with uncontrolled intercurrent illness including, but not limited to: ongoing
or active infection (except for uncomplicated urinary tract infection), uncontrolled
interstitial lung disease, symptomatic congestive heart failure, or psychiatric
illness/social situations that would limit compliance with study requirements
• Treatment with strong CYP2C8 or CYP3A4 inhibitors or inducers within 14 days or 5
drug-elimination half-lives, whichever is longer, prior to registration
• Women who are unwilling to discontinue nursing
Endometrial Serous Adenocarcinoma, Uterine Corpus Carcinosarcoma, Corpus Uteri, Uterus
A Trial of Robotic Versus Open Hysterectomy Surgery in Cervix Cancer (ROCC)
This is a randomized controlled trial to compare survival for patients who undergi robotic
assisted laparoscopy versus open radical hysterectomy and lymph node assessment for the
treatment of early stage cervical cancer.
Stephen Rose, MD
Female
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04831580
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Inclusion Criteria:
1. Patient must have histologically confirmed adenocarcinoma (usual/classic/NOS),
squamous cell carcinoma, adenosquamous carcinoma (Including glassy cell)
2. Patient must be FIGO Stage IA2, IBI, IB2 (2018 staging) without evidence of definitive
parametrial, vaginal, nodal or distant metastases on exam or imaging. Patients with
tumor size less than or equal to 4 cm confirmed on MRI prior to randomization are
eligible.
3. Patient must have uterine size <12 cm AND felt to be appropriate for vaginal delivery
of the specimen per investigator.
4. Patient must be suitable surgical candidate with preoperative assessments such as labs
and EKG performed per institutional standard.
5. Patient must be age 18 years or older.
6. Patient must have ECOG performance status 0-1.
7. Patient must have a negative urine pregnancy test within 30 days of surgery in
pre-menopausal women.
8. Patient must have signed an approved informed consent and authorization permitting the
release of personal health information.
Exclusion Criteria:
1. Patients with any tumor histology other than those listed above, specifically
excluding the following histologies: neuroendocrine, other adenocarcinoma (gastric
type, endometrioid, clear cell, serous, signet ring, minimal deviation)
2. Patients with FIGO stage 1A1, IB3, II-IV (2018 staging).
3. Patient with inability to receive an MRI.
4. Patients with a tumor size greater than 4cm or on MRI confirmed prior to randomization
are excluded. Patients with definite evidence of vaginal/parametrial involvement on
MRI are excluded; if MRI findings are not definitive, then clinical examination must
also not reveal parametrial or vaginal extension).
5. Patients with evidence of metastatic disease (imaging or histologically positive lymph
nodes).
6. Patients with a history of prior pelvic or abdominal radiotherapy.
7. Patients with a prior malignancy < 5 years from enrollment with the exception of
non-melanoma skin cancer.
8. Patients who are unable to withstand prolonged lithotomy or steep trendelenberg.
9. Patient compliance and geographic proximity that do not allow adequate follow-up.
10. Patients with poorly controlled HIV with CD4 counts <500.
Study of Efficacy and Safety of Inhaled Treprostinil in Subjects With Idiopathic Pulmonary Fibrosis (TETON)
Study RIN-PF-301 is designed to evaluate the superiority of inhaled treprostinil against
placebo for the change in absolute forced vital capacity (FVC) from baseline to Week 52.
Stephen Halliday
All
40 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04708782
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Inclusion Criteria:
1. Subject gives voluntary informed consent to participate in the study.
2. Subject is ≥40 years of age, inclusive, at the time of signing informed consent.
3. The subject has a diagnosis of IPF based on the 2018 ATS/ERS/JRS/ALAT Clinical
Practice Guideline (Raghu 2018) and confirmed by central review of high-resolution
computed tomography (HRCT) (performed within the previous 12 months), and if
available, surgical lung biopsy.
4. FVC ≥45% predicted at Screening.
5. Subjects on pirfenidone or nintedanib must be on a stable and optimized dose for ≥30
days prior to Baseline. Concomitant use of both pirfenidone and nintedanib is not
permitted.
6. Women of childbearing potential must be non-pregnant (as confirmed by a urine
pregnancy test at Screening and Baseline) and non-lactating, and will abstain from
intercourse (when it is in line with their preferred and usual lifestyle) or use 2
medically acceptable, highly effective forms of contraception for the duration of the
study, and at least 30 days after discontinuing study drug.
7. Males with a partner of childbearing potential must use a condom for the duration of
treatment and for at least 48 hours after discontinuing study drug.
8. In the opinion of the Investigator, the subject is able to communicate effectively
with study personnel, and is considered reliable, willing, and likely to be
cooperative with protocol requirements, including attending all study visits.
Exclusion Criteria:
1. Subject is pregnant or lactating.
2. Subject has primary obstructive airway physiology: FEV1/FVC <0.70 at Screening.
3. The subject has shown intolerance or significant lack of efficacy to a prostacyclin or
prostacyclin analogue that resulted in discontinuation or inability to effectively
titrate that therapy.
4. The subject has received any PAH-approved therapy, including prostacyclin therapy
(epoprostenol, treprostinil, iloprost, or beraprost; except for acute vasoreactivity
testing), IP receptor agonists (selexipag), endothelin receptor antagonists,
phosphodiesterase type 5 inhibitors (PDE5-Is), or soluble guanylate cyclase
stimulators within 60 days prior to Baseline. As needed use of a PDE5-I for erectile
dysfunction is permitted, provided no doses are taken within 48 hours of any
study-related efficacy assessments.
5. Use of any of the following medications: azathioprine (AZA), cyclosporine,
mycophenolate mofetil, tacrolimus, oral corticosteroids (OCS) >20 mg/day or the
combination of OCS+AZA+N-acetylcysteine within 30 days prior to Baseline;
cyclophosphamide within 60 days prior to Baseline; or rituximab within 6 months prior
to Baseline.
6. The subject is receiving >10 L/min of oxygen supplementation by any mode of delivery
at rest at Baseline.
7. Exacerbation of IPF or active pulmonary or upper respiratory infection within 30 days
prior to Baseline. Subjects must have completed any antibiotic or steroid regimens for
treatment of the infection or acute exacerbation more than 30 days prior to Baseline
to be eligible. If hospitalized for an acute exacerbation of IPF or a pulmonary or
upper respiratory infection, subjects must have been discharged more than 90 days
prior to Baseline to be eligible.
8. Uncontrolled cardiac disease, defined as myocardial infarction within 6 months prior
to Baseline or unstable angina within 30 days prior to Baseline.
9. In the opinion of the Investigator, the subject has any condition that would interfere
with the interpretation of study assessments or would impair study participation or
cooperation.
10. Use of any other investigational drug/device or participation in any investigational
study in which the subject received a medical intervention (ie, procedure, device,
medication/supplement) within 30 days prior to Screening. Subjects participating in
non-interventional, observational, or registry studies are eligible.
11. Life expectancy <6 months due to IPF or a concomitant illness.
12. Acute pulmonary embolism within 90 days prior to Baseline.
Phase 2 Study of DKN-01 in Colorectal Cancer (DeFianCe)
This is a Phase 2 randomized, open-label, two-part, multicenter study with a safety run-in to
evaluate efficacy and safety of DKN-01 plus FOLFIRI/FOLFOX and bevacizumab versus standard of
care (SOC) [FOLFIRI/FOLFOX and bevacizumab] as second-line treatment of advanced CRC
patients.
Dustin Deming, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05480306
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Adult patients with advanced CRC with measurable disease (RECIST v1.1) who have
radiographically progressed during or following one line of systemic treatment will be
enrolled in the study.
Inclusion Criteria:
Patients meeting all of the following criteria will be considered eligible for study entry:
1. Disease progression following first-line systemic therapy with any
fluoropyrimidine-based regimen for advanced disease (except FOLFOXIRI, see exclusion
criteria).
• Patients may have received prior neoadjuvant or adjuvant therapy which could have
included irinotecan or oxaliplatin. If progression has occurred within 12 months from
last dose of neoadjuvant or adjuvant treatment, this regimen will be considered as the
one line of systemic therapy for advanced disease.
• If assigned to receive FOLFIRI, patient may have received no prior irinotecan as
part of first-line systemic therapy.
• If assigned to receive FOLFOX, patient may have received no prior oxaliplatin as
part of first line systemic therapy.
• Prior treatment with an anti-VEGF or anti-EGFR therapy is allowed as first-line
and/or maintenance systemic therapy.
2. Able to provide written informed consent for any study specific procedures.
3. One or more tumors measurable on radiographic imaging as defined by RECIST 1.1
4. Sufficient tumor tissue for mandatory pre-treatment evaluation (fresh biopsy
[preferred], or archived tissue block specimen).
5. ECOG performance status ≤1 within 7 days of first dose of study drug. Acceptable
liver, renal, hematologic, and coagulation function
6. Females of childbearing potential and male partners of female patients must agree to
use adequate contraception during the study and for 6 months after their last dose of
study drug
Exclusion Criteria:
Patients meeting any of the following criteria are not eligible for study entry:
1. Diagnosis of Microsatellite instability-high (MSI-H)/mismatch repair-deficient (dMMR)
and/or BRAF V600E mutation positive colorectal cancer.
2. Prior therapy with an anti-DKK1, FOLFOXIRI, PD-1, anti-PD-L1, anti-PD-L-2 or any other
antibody or drug specifically targeting T-cell co-stimulation or coinhibitory
checkpoint.
3. Systemic anti-cancer therapy within 28 days prior to first dose of study drug.
4. Major surgery within 28 days prior to first dose of study drug.
5. Prior radiation therapy within 14 days prior to first dose of study drug.
6. Active leptomeningeal disease or uncontrolled brain metastases.
7. Any active cancer ≤ 2 years before first dose of study drug with the exception of
cancer for this study.
8. New York Heart Association Class III or IV cardiac disease, myocardial infarction
within the past 6 months, or unstable arrhythmia.
9. Fridericia-corrected QT interval (QTcF) > 470 msec (female) or history of congenital
long QT syndrome.
10. Active, uncontrolled bacterial, viral, or fungal infections, within 14 days of study
entry requiring systemic therapy.
11. Serious nonmalignant disease
12. Pregnant or nursing.
13. History of osteonecrosis of the hip or have evidence of structural bone abnormalities
in the proximal femur on MRI scan that are symptomatic and clinically significant.
14. Known osteoblastic bony metastasis.
15. Major surgery 28 days prior to study entry.
16. Prior radiation therapy within 14 days prior to study entry.
17. Significant allergy to a pharmaceutical therapy that, in the opinion of the
Investigator, poses an increased risk to the patient.
18. Active substance abuse.
19. Known dihydropyrimidine dehydrogenase deficiency.
20. Administration of a live vaccine within 28 days before first dose of study drug
Colorectal Cancer, Colorectal Adenocarcinoma, Colo-rectal Cancer, Colorectal Cancer Metastatic, Colon, Rectum, Colon and Rectum
This study will evaluate the effectiveness and safety of an investigational product (IP),
intravenous (IV) ganaxolone, to treat participants with status epilepticus (SE).
Aaron Struck, Medical Student
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04391569
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Inclusion Criteria:
1. Participant, participant's parent, guardian, or legal authorized representative (LAR)
must provide signed of informed consent/assent, and once capable (per institution
guidelines), there must be documentation of consent/assent by the participant
demonstrating they are willing and aware of the investigational nature of the study
and related procedures. Where allowed by law, where the participant lacks the capacity
to make informed decisions regarding his/her medical treatment options, the treating
clinician may follow their deferred consenting practices. The clinician will make the
final decision based on the best interests of the particiapant.
2. Male or females 12 years of age and older at the time of the first dose of IP
3. SE meeting the following criteria:
a. A diagnosis of SE with or without prominent motor features based on clinical and
EEG findings:
i. Diagnosis is established by:
• For SE with prominent motor features: Clinical and EEG seizure activity
indicative of convulsive, myoclonic or focal motor SE.
• For SE without prominent motor features (nonconvulsive SE): Appropriate clinical
features and an EEG indicative of non-convulsive status epilepticus (NCSE)
ii. For any type of SE:
• At least 6 minutes of cumulative seizure activity over a 30-minute period within
the hour before IP initiation, AND
• Seizure activity during the 30 minutes immediately prior to IP initiation
b. The treating clinician(s) anticipate that IV anesthesia is likely to be the
next treatment for SE that persists following initiation of IP
4. Participants must have received any two or more of the following agents for treatment
of the current episode of SE administered at an adequate dose and for a sufficient
duration, in the judgment of the investigator, to demonstrate efficacy
• Benzodiazepines,
• IV Fosphenytoin/phenytoin,
• IV Valproic acid,
• IV Levetiracetam,
• IV Lacosamide,
• IV Brivaracetam, or
• IV Phenobarbital
5. Body mass index (BMI) < 40 or, if BMI is not able to be calculated at screening,
participant is assessed by investigator as not morbidly obese
Exclusion Criteria:
1. Life expectancy of less than 24 hours
2. Anoxic brain injury or an uncorrected rapidly reversable metabolic condition as the
primary cause of SE (e.g., hypoglycemia < 50 milligram per deciliter [mg/dL] or
hyperglycemia > 400 mg/dL)
3. Participants who have received high-dose IV anesthetics (e.g., midazolam, propofol,
thiopental, or pentobarbital) during the current episode of SE for more than 18 hours,
or who continue to have clinical or electrographic evidence of persistent seizures
while receiving high-dose IV anesthetics.
4. Clinical condition or advance directive that would NOT permit use of IV anesthesia
5. Participants known or suspected to be pregnant
6. Participants with known allergy or sensitivity to progesterone or allopregnanolone
medications/supplements
7. Receiving a concomitant IV product containing Captisol®
8. Known or suspected hepatic insufficiency or hepatic failure leading to impaired
synthetic liver function.
9. Known or suspected stage 3B (moderate to severe; estimated glomerular filtration rate
[eGFR] 44-30 milliliter/minutes/1.73-meter square [mL/min/1.73m^2]), stage 4 (severe;
eGFR 29-15 mL/min/1.73m^2), or stage 5 (kidney failure; eGFR < 15 mL/min/1.73m^2 or
dialysis) kidney disease
10. Use of an investigational product for which less than 30 days or 5 half-lives have
elapsed from the final product administration. Participation in a non-interventional
clinical study does not exclude eligibility.
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