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within category "Congenital & Chromosomal Abnormalities"
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Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This is a long-term follow-up safety and efficacy study of participants in clinical trials
for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
Participants will roll over from their respective previous (parent) study into this long-term
study for continuous monitoring of safety as well as monitoring of continued efficacy and
durability of response to onasemnogene abeparvovec-xioi treatment.
Jennifer Kwon, MD
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT04042025
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Inclusion Criteria:
• Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement
therapy in a Novartis Gene Therapies-sponsored clinical study
• Participant/parent/legal guardian willing and able to complete the informed consent
process and comply with study procedures and visit schedule
Exclusion Criteria:
• Parent/legal guardian unable or unwilling to participate in the long-term follow-up
safety study
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA, Spinal muscular atrophy, Other, Congenital & Chromosomal Abnormalities
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS) (POPS or POP02)
The study investigators are interested in learning more about how drugs, that are given to
children by their health care provider, act in the bodies of children and young adults in
hopes to find the most safe and effective dose for children. The primary objective of this
study is to evaluate the PK of understudied drugs currently being administered to children
per SOC as prescribed by their treating provider.
Maria Stanley
All
0 Years to 20 Years old
NA
This study is also accepting healthy volunteers
NCT04278404
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Inclusion Criteria:
1. Participant is < 21 years of age
2. Parent/ Legal Guardian/ Adult Participant can understand the consent process and is
willing to provide informed consent/HIPAA:
3. (a) Participant is receiving one or more of the study drugs of interest at the time of
enrollment or (b) Participant is NOT receiving one or more of the study drugs of
interest but is SARS-COV-2 positive within 60 days prior to enrollment
Exclusion Criteria:
1. Participant has a known pregnancy
Below exclusion criteria apply only to:
Participants receiving one or more of the study drugs of interest at the time of
enrollment, DOI administration or PK sampling: (Refer to DOI specific appendices for
details on enrollment cohort specifications and additional eligibility criteria)
2. Has had intermittent dialysis within previous 24 hours
3. Has had a kidney transplant within previous 30 days
4. Has had a liver transplant within previous 1 year
5. Has had a stem cell transplant within previous 1 year
6. Has had therapeutic hypothermia within previous 24 hours
7. Has had plasmapheresis within the previous 24 hours
8. Has a Ventricular Assist Device
9. Has any condition which would make the participant, in the opinion of the
investigator, unsuitable for the study
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension, Pain, Hyperphosphatemia, Primary Hyperaldosteronism, Edema, Hypokalemia, Heart Failure, Hemophilia, Menorrhagia, Insomnia, Pneumonia, Skin Infection, Arrythmia, Asthma in Children, Bronchopulmonary Dysplasia, Adrenal Insufficiency, Fibrinolysis, Hemorrhage, Attention Deficit Hyperactivity Disorder, Multisystem Inflammatory Syndrome in Children (MIS-C), Kawasaki Disease, Coagulation Disorder, Down Syndrome, Other
CHaractErizing CFTR Modulated Changes in Sweat Chloride and Their Association With Clinical Outcomes (CHEC-SC)
This is a multicenter, cross-sectional, cohort study which will collect contemporary sweat
chloride (SC) values from approximately 5000 Cystic Fibrosis (CF) patients prescribed and
currently receiving commercially approved Cystic Fibrosis transmembrane conductance regulator
(CFTR) modulator therapies.
Hara Levy, MD
All
4 Months and over
NA
This study is NOT accepting healthy volunteers
NCT03350828
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Inclusion Criteria:
1. Written informed consent (and assent when applicable) obtained from subject or
subject's legal representative
2. Enrolled in the CFFPR
3. Male or female ≥ 4 months of age on day of study visit
4. Diagnosis of CF.
5. Current treatment with a prescribed commercially approved CFTR modulator for at least
90 days prior to enrollment
6. Able to perform the testing and procedures required for this study, as judged by the
investigator
Additional Inclusion Criteria for CHEC-PKPD Sub-Study:
1. Male or female ≥ 6 years of age on day of study visit.
2. Current treatment with elexacaftor/tezacaftor/ivacaftor for at least 90 days prior to
enrollment.
3. Last dose of elexacaftor/tezacaftor/ivacaftor taken at least 24hours and last dose of
ivacaftor taken at least 12 hours prior to trough blood draw on day of visit.
Exclusion Criteria:
1. Presence of a condition or abnormality that, in the opinion of the Investigator, would
compromise the safety of the patient or the quality of the data
2. Currently enrolled in an investigational trial (including open-label follow-on studies
and Early Access Programs (EAP) of an agent expected to have an impact on sweat
chloride (refer to current list provided on study website)
Cystic Fibrosis, Respiration Disorders [C08], Other
APL-101 Study of Subjects With NSCLC With c-Met EXON 14 Skip Mutations and c-Met Dysregulation Advanced Solid Tumors (SPARTA)
To assess:
- efficacy of APL-101 as monotherapy for the treatment of NSCLC harboring MET Exon 14
skipping mutations, NSCLC harboring MET amplification, solid tumors harboring MET
amplification, solid tumors harboring MET fusion, primary CNS tumors harboring MET
alterations, solid tumors harboring wild-type MET with overexpression of HGF and MET
- efficacy of APL-101 as an add-on therapy to EGFR inhibitor for the treatment of NSCLC
harboring EGFR activating mutations and developed acquired resistance with MET
amplification and disease progression after documented CR or PR with 1st line EGFR
inhibitors (EGFR-I)
Mark Burkard, MD, PhD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03175224
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Major
Inclusion Criteria:
1. Men and women 18 years of age or older.
2. 9 cohorts will be enrolled:
• Cohort A1 / Exon 14 NSCLC MET inhibitor naive in first line: Histologically or
cytologically confirmed NSCLC with Exon 14 skipping mutations; all histologies;
unresectable or metastatic disease (Stage 3b/4); treatment-naive subjects in
first line; not received any MET inhibitor and no known MET kinase inhibitor
resistance mutations
• Cohort A2 / Exon 14 NSCLC •MET inhibitor naïve: Histologically or cytologically
confirmed NSCLC with Exon 14 skipping mutations; all histologies; unresectable or
metastatic disease (Stage 3b/4); pretreated subjects refractory to or intolerant
of standard therapies with no more than three lines of prior therapy in the
unresectable or metastatic setting; not received any MET inhibitor and no known
MET kinase inhibitor resistance mutations
• Cohort B / Exon 14 NSCLC MET inhibitor experienced: ENROLLMENT COMPLETED
• Cohort C / MET amplification basket tumor types excluding primary CNS tumors: Any
solid tumor type regardless of histology excluding primary CNS tumors, with MET
amplification; unresectable or metastatic disease, refractory to or intolerant of
standard therapies, or refused standard therapies, or if therapy was unavailable
or unfeasible, with no more than 3 prior lines of therapy in the unresectable or
metastatic setting; not received any MET inhibitor and no known MET kinase
inhibitor resistance mutations
• Cohort C1 / MET amplification and wild-type EGFR NSCLC: NSCLC regardless of
histology, harboring MET amplification and wild-type EGFR; unresectable or
metastatic disease, previously untreated or treated with no more than 3 prior
lines of therapy in the unresectable or metastatic setting; not received any MET
inhibitor and no known MET kinase inhibitor resistance mutations
• Cohort C2 / EGFR positive NSCLC with acquired MET amplification (APL-101 Add-on
Therapy): Unresectable or metastatic NSCLC regardless of histology, harboring
EGFR activating mutations with acquired MET-Amplification as resistance mechanism
to the EGFR-I; developed resistance to first-line EGFR-inhibitor therapy after an
initial response (documented PR for at least 12 weeks); radiological
documentation of disease progression per RECIST on first-line EGFR inhibitor
therapy; currently on an EGFR-inhibitor therapy and agrees to receive APL-101 as
an add-on therapy during the study; no history of interstitial lung disease
(ILD)/pneumonitis, Grade ≥3 liver toxicity or QT prolongation with EGFR-I
therapy; not received any MET inhibitor and no known MET kinase inhibitor
resistance mutations
• Cohort D / MET fusion basket tumor types excluding primary CNS tumors: any solid
tumor type regardless of histology excluding primary CNS tumors; unresectable or
metastatic disease, refractory to or intolerant of standard therapies, or refused
standard therapies, or if therapy was unavailable or unfeasible, with no more
than 3 prior lines of therapy in the unresectable or metastatic setting; not
received any MET inhibitor and no known MET kinase inhibitor resistance mutations
• Cohort E / Primary CNS tumors with MET alterations: subjects with primary CNS
tumors who meet inclusion criteria of MET dysregulations defined as single or
co-occurred MET fusion including PTPRZ1-MET (ZM) fusion, MET Exon 14 skipping
mutations, or MET amplification; refractory to or intolerant of standard
therapies, or refused standard therapies, or if therapy was unavailable or
unfeasible, with no more than 3 prior lines of therapy in the unresectable or
metastatic setting; not received any MET inhibitor and no known MET kinase
inhibitor resistance mutations; neurological symptoms controlled on a
stable/decreasing dose of steroids for at least 2 weeks before C1D1
• Cohort F / Basket tumor types harboring wild-type MET with over-expression of HGF
and MET: any solid tumor type regardless of histology harboring wild-type MET
with overexpression of HGF and MET; Unresectable or metastatic disease,
refractory to or intolerant of standard therapies, or refused standard therapies,
or if therapy was unavailable or unfeasible, with no more than 3 prior lines of
therapy in the unresectable or metastatic setting; not received any MET inhibitor
and no known MET kinase inhibitor resistance mutations
3. Treated or untreated asymptomatic parenchymal CNS disease or leptomeningeal disease is
allowed.
4. Presence of ≥1 measurable lesion (scan done ≤28 days of C1D1) to serve as target
lesion according to relevant criteria
5. ECOG performance status of 0-1. For subjects with primary CNS tumors, KPS score ≥70.
6. Acceptable organ function
7. For all prior anticancer treatment, a duration of 30 days or 5 half-lives of the
agents used, whichever is shorter, must have elapsed, and any encountered toxicity
must have resolved to levels meeting all the other eligibility criteria prior to the
first dose of study treatment. Palliative radiotherapy to non-target lesions should be
completed within 2 weeks prior to APL-101 administration.
8. Adequate cardiac function
9. Women of child-bearing potential must have a negative serum or Beta-hCG at screening
or evidence of surgical sterility or evidence of post-menopausal status
10. No planned major surgery within 4 weeks of first dose of APL-101
11. Expected survival (life expectancy) ≥ 3 months from C1D1
12. Provision of sample; e.g. archival or a fresh tumor biopsy sample (if safe and
feasible) either from the primary or a metastatic site) or liquid biopsy sample (if
tumor tissue is insufficient or lacking, and approved by the sponsor) is required for
prospective central lab confirmation for study entry (subjects with previously
confirmed molecular status by the Sponsor designated central lab or FDA approved NGS
based MET testing may be exempted, subjected to Sponsor approval.
Major
Exclusion Criteria:
1. Hypersensitivity to APL-101, excipients of the drug product, or other components of
the study treatment regimen.
2. Known actionable mutation/gene rearrangement of EGFR (except for NSCLC subjects in
Cohort C and C-2), ALK, ROS1, RET, NTRK, KRAS, and BRAF.
3. Use or intended use of any other investigational product, including herbal
medications, through Study Treatment Termination.
4. Active uncontrolled systemic bacterial, viral, or fungal infection or clinically
significant, active disease process, which in the opinion of the investigator makes
the risk: benefit unfavorable for the participation of the trial.
5. Life-threatening illness, significant organ system dysfunction or comorbid conditions,
or other reasons that, in the investigator's opinion, could compromise the subject's
safety or the integrity of the study outcomes, or interfere with the absorption or
metabolism of APL-101.
6. Unstable angina or myocardial infarction within 1 year prior to first dose of APL-101,
symptomatic or unstable arrhythmia requiring medical therapy, history of congenital
prolonged QT syndrome, prolonged QT interval corrected by Fridericia formula (QTcF) at
screening, or concurrent treatment with a medication that is a known risk for
prolonging the QT interval. Chronic controlled atrial fibrillation is not excluded.
7. Historical seropositive results consistent with active infection for hepatitis C virus
(HCV) or hepatitis B virus (HBV) with high viral loads not actively managed with
antiviral therapy and human immunodeficiency virus (HIV) positive subjects who are not
clinically stable or controlled on their medication (asymptomatic subjects with CD4+
T-cell (CD4+) counts ≥ 350 cells/μL and have not had an opportunistic infection within
the past 12 months prior to first dose of APL-101 would be eligible for study entry.
If history is unclear, relevant test(s) at Screening will be required to confirm
eligibility.
8. Known significant mental illness or other conditions such as active alcohol or other
substance abuse that, in the opinion of the investigator, predisposes the subject to
high risk of noncompliance with the protocol treatment or assessments.
9. Unable to swallow orally administered medication whole.
10. Impairment of gastrointestinal function or gastrointestinal disease that may
significantly alter drug absorption
11. Women who are breastfeeding
12. History of another malignancy within 3 years prior to C1D1. A subject with the
following malignancies is allowed if considered cured or unlikely to recur within 3
years:
1. Carcinoma of the skin without melanomatous features.
2. Curatively treated cervical carcinoma in situ.
3. Bladder tumors considered superficial such as noninvasive (T1a) and carcinoma in
situ (T1s), thyroid papillary cancer with prior treatment, prostate cancer which
has been surgically or medically treated and not likely to recur within 3 years.
13. Subjects who are unable or unwilling to discontinue excluded medications (drugs with
known QTc risk and known strong cytochrome P450 [CYP]3A4 inducer and/or strong
inhibitors) for at least 5 half-lives prior to first dose of study drug. Subjects may
qualify if such medication(s) can be safely replaced with alternate medications with
less risk of drug-drug interaction.
14. Subjects with active COVID-19 infection.
15. Symptomatic and/or neurologically unstable CNS metastases, or who require an increase
in steroid dose to control CNS disease. Subjects who have been receiving a stable
steroid dose for at least 2 weeks prior to C1D1 may be allowed.
Solid Tumors, Advanced Cancer, Renal Cancer, Gastric Cancer, Gastroesophageal Junction Adenocarcinoma, NSCLC, Lung Cancer, Brain Tumor, Glioblastoma Multiforme, EGFR Gene Mutation, MET Amplification, HGF, Thyroid Cancer, Pancreatic Cancer, Colon Cancer, MET Alteration, MET Fusion, Exon 14 Skipping, 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, Melanoma, Skin, 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
A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia
This phase III trial studies how well blinatumomab works in combination with chemotherapy in
treating patients with newly diagnosed, standard risk B-lymphoblastic leukemia or
B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as
blinatumomab, may induce changes in the body's immune system and may interfere with the
ability of cancer cells to grow and spread. Chemotherapy drugs, such as vincristine,
dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine,
mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to
stop the growth of cancer cells, either by killing the cells, by stopping them from dividing,
or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate.
Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving
blinatumomab and combination chemotherapy may work better than combination chemotherapy alone
in treating patients with B-ALL. This trial also assigns patients into different chemotherapy
treatment regimens based on risk (the chance of cancer returning after treatment). Treating
patients with chemotherapy based on risk may help doctors decide which patients can best
benefit from which chemotherapy treatment regimens.
Kenneth Desantes, M.D.
All
365 Days to 31 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03914625
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Inclusion Criteria:
• All B-ALL patients must be enrolled on APEC14B1 and consented to Eligibility Screening
(Part A) prior to treatment and enrollment on AALL1731. APEC 14B1 is not a requirement
for B-LLy patients. B-LLy patients may directly enroll on AALL1731.
• Age at diagnosis:
• Patients must be >= 365 days and < 10 years of age (B-ALL patients without DS).
• Patients must be >= 365 days and =< 31 years of age (B-ALL patients with DS).
• Patients must be >= 365 days and =< 31 years of age (B-LLy patients with or
without DS).
• B-ALL patients without DS must have an initial white blood cell count < 50,000/uL
(performed within 7 days prior to enrollment).
• B-ALL patients with DS are eligible regardless of the presenting white blood cell
count (WBC) (performed within 7 days prior to enrollment).
• Patient has newly diagnosed B-cell ALL, with or without Down syndrome: > 25% blasts on
a bone marrow (BM) aspirate;
• OR if a BM aspirate is not obtained or is not diagnostic of B-ALL, the diagnosis
can be established by a pathologic diagnosis of B-ALL on a BM biopsy;
• OR a complete blood count (CBC) documenting the presence of at least 1,000/uL
circulating leukemic cells;
• OR patient has newly diagnosed B-cell LLy Murphy stages I or II, with or without
Down syndrome.
• Note: For B-LLy patients with tissue available for flow cytometry, the criterion
for diagnosis should be analogous to B-ALL. For tissue processed by other means
(i.e., paraffin blocks), the methodology and criteria for immunophenotypic
analysis to establish the diagnosis of B-LLy defined by the submitting
institution will be accepted (diagnostic biopsy for B-LLy must be performed
within 14 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:
• Patient must not have secondary ALL that developed after treatment of a prior
malignancy with cytotoxic chemotherapy. Note: patients with Down syndrome with a prior
history of transient myeloproliferative disease (TMD) are not considered to have had a
prior malignancy. They would therefore be eligible whether or not the TMD was treated
with cytarabine.
• With the exception of steroid pretreatment or the administration of intrathecal
cytarabine, patients must not have received any prior cytotoxic chemotherapy for
either the current diagnosis of B ALL or B LLy or for any cancer diagnosed prior to
initiation of protocol therapy on AALL1731.
• For patients receiving steroid pretreatment, the following additional exclusion
criteria apply:
• Non-DS B-ALL patients must not have received steroids for more than 24 hours in
the 2 weeks prior to diagnosis without a CBC obtained within 3 days prior to
initiation of the steroids.
• DS and non-DS B-LLy patients must not have received > 48 hours of oral or IV
steroids within 4 weeks of diagnosis.
• Patients who have received > 72 hours of hydroxyurea within 1 week (7 days) prior to
the start of systemic protocol therapy.
• B-ALL patients who do not have sufficient diagnostic bone marrow submitted for
APEC14B1 diagnostic testing and who do not have a peripheral blood sample submitted
containing > 1,000/uL circulating leukemia cells.
• Patient must not have acute undifferentiated leukemia (AUL).
• Non-DS B-ALL patients with central nervous system [CNS]3 leukemia (CNS status must be
known prior to enrollment).
• Note: DS patients with CNS3 disease are eligible but will be assigned to the
DS-High B-ALL arm. CNS status must be determined based on a sample obtained prior
to administration of any systemic or intrathecal chemotherapy, except for steroid
pretreatment.
• Non-DS B-ALL patients with testicular leukemia. (Note: DS patients with testicular
disease are eligible but will be assigned to the DS-High B-ALL arm).
• For LLy patients, the following additional exclusion criteria apply:
• T-Lymphoblastic Lymphoma.
• Morphologically unclassifiable lymphoma.
• Absence of both B-cell and T-cell phenotype markers in a case submitted as
lymphoblastic lymphoma.
• CNS positive disease or testicular involvement.
• M2 (5% •25% blasts) or M3 (> 25% blasts) marrow.
• Patients with known Charcot-Marie-Tooth disease.
• Patients with known MYC translocation associated with mature (Burkitt) B-cell ALL,
regardless of blast immunophenotype.
• Patients requiring radiation at diagnosis.
• 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.
B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Down Syndrome, Non-Hodgkin's Lymphoma, Lymphoid Leukemia, Leukemia, other, Leukemia, Lymphoma
Project: Every Child for Younger Patients With Cancer
This study gathers health information for the Project: Every Child for younger patients with
cancer. Gathering health information over time from younger patients with cancer may help
doctors find better methods of treatment and on-going care.
Kenneth Desantes, M.D.
All
up to 25 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02402244
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Inclusion Criteria:
• Enrollment must occur within 6 months of initial disease presentation OR within 6
months of refractory disease, disease progression, disease recurrence, second or
secondary malignancy, or post-mortem
• Patients previously enrolled on ACCRN07 are eligible to enroll on Tracking Outcome,
Registry and Future Contact components of APEC14B1 any time after they reach age of
majority
• Patients with a known or suspected neoplasm that occurs in the pediatric, adolescent
or young adult populations are eligible for enrollment as follows:
• All cancer cases with an International Classification of Diseases for Oncology
(ICD-O) histologic behavior code of one "1" (borderline), two "2" (carcinoma in
situ) or three "3" (malignant)
• All neoplastic lesions of the central nervous system regardless of behavior,
i.e., benign, borderline or malignant
• The following other benign/borderline conditions:
• Mesoblastic nephroma
• Teratomas (mature and immature types)
• Myeloproliferative diseases including transient myeloproliferative disease
• Langerhans cell histiocytosis
• Lymphoproliferative diseases
• Desmoid tumors
• Gonadal stromal cell tumors
• Neuroendocrine tumors including pheochromocytoma
• Melanocytic tumors, except clearly benign nevi
• Ganglioneuromas
• Subjects must be =< 25 years of age at time of original diagnosis, except for patients
who are being screened specifically for eligibility onto a COG (or COG participating
National Clinical Trials Network [NCTN]) therapeutic study, for which there is a
higher upper age limit
• All patients or their parents or legally authorized representatives must sign a
written informed consent and agree to participate in at least one component of the
study; parents will be asked to sign a separate consent for their own biospecimen
submission
• If patients or their parents or legally authorized representatives have not
signed the Part A subject consent form at the time of a diagnostic bone marrow
procedure, it is recommended that they initially provide consent for drawing
extra bone marrow using the Consent for Collection of Additional Bone Marrow;
consent using the Part A subject consent form must be provided prior to any other
procedures for eligibility screening or banking under APEC14B1
Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neuroendocrine Neoplasm, Stromal Neoplasm, 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, Melanoma, Skin, 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, Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Leukemia, other, Other Hematopoietic, Unknown Sites, Ill-Defined Sites, Any Site, Anal, Bladder, Brain/Central Nervous System, Colon and Rectum, Endocrine cancers, Gastrointestinal cancers, other, Genitourinary cancers, other, Head and Neck, Hematologic cancers, other, Leukemia, Lymphoma, Melanoma/Skin cancer, Sarcoma, Uterus
This research trial studies kidney tumors in younger patients. Collecting and storing samples
of tumor tissue, blood, and urine from patients with cancer to study in the laboratory may
help doctors learn more about changes that occur in deoxyribonucleic acid (DNA) and identify
biomarkers related to cancer.
Kenneth Desantes, M.D.
All
up to 29 Years old
N/A
This study is NOT accepting healthy volunteers
NCT00898365
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Inclusion Criteria:
• Patients with the first occurrence of any tumor of the kidney identified on CT scan or
MRI are eligible for this study; histologic diagnosis is not required prior to
enrollment but is required for all patients once on study
• Eligible tumors include (but are not limited to):
• Nephroblastic tumors
• Nephroblastoma (Wilms' tumor) (favorable histology, anaplasia [diffuse,
focal])
• Nephrogenic rests and nephroblastomatosis
• Cystic nephroma and cystic partially differentiated nephroblastoma
• Metanephric tumors (metanephric adenoma, metanephric adenofibroma,
metanephric stromal tumor)
• Mesoblastic nephroma (cellular, classic, mixed)
• Clear cell sarcoma
• Rhabdoid tumor (any malignant rhabdoid tumor occurring outside the central
nervous system [CNS])
• Renal epithelioid tumors of childhood (papillary renal cell carcinoma, medullary
renal cell carcinoma, renal tumors associated with Xp11.2 translocations,
oncocytic renal neoplasms after neuroblastoma)
• Angiolipoma
• Ossifying renal tumor of infancy
• Patients with the first occurrence of the following tumors are also eligible:
• Extrarenal nephroblastoma or extrarenal neprogenic rests
• Malignant rhabdoid tumor occurring anywhere outside the central nervous system
• Required specimens, reports, forms, and copies of imaging studies must be available or
will become available for submission and the institution must intend on submitting
them as described in the protocol procedures
• For ALL patients, (with exception of bilateral, bilaterally predisposed, multicentric,
or unilateral tumor in solitary kidney planning to enroll without biopsy***), the
following submissions are required:
• A complete set of recut hematoxylin and eosin (H & E) slides (including from
sampled lymph nodes, if patient had upfront nephrectomy)
• * Tissue must be from diagnosis, prior to any renal tumor directed
chemotherapy or radiation (only exception is for presumed favorable
histology Wilms tumor [FHWT] patients discovered to have diffuse anaplastic
Wilms tumor [DAWT] at delayed nephrectomy and plan to enroll at delayed
nephrectomy)
• Representative formalin-fixed paraffin-embedded tissue block or if a block is
unavailable, 10 unstained slides from a representative block of tumor, if
available.
• Tissue must be from diagnosis, prior to any renal tumor directed
chemotherapy or radiation (only exception is for presumed FHWT patients
discovered to have DAWT at delayed nephrectomy and plan to enroll at delayed
nephrectomy)
• Institutional pathology report, Specimen Transmittal Form, and Pre-Treatment
Pathology Checklist
• Copies of images and institutional reports of CT and/or MRI abdomen and pelvis,
and Pre Treatment Imaging Checklist
• Copies of images and institutional report of chest CT for all malignant tumors
• Institutional surgical report(s) and Pre-Treatment Surgical Checklist
• CRFs: Staging Checklist and Metastatic Disease Form (if metastatic disease is
noted on imaging)
• Patients with bilateral, bilaterally predisposed, multicentric, or
unilateral tumor in solitary kidney planning to enroll without biopsy via
imaging only •these patients will not have central review or have a risk
assignment issued, but may contribute to specimen banking for future
research. However, if biopsy is done, tissue must be submitted as for other
renal tumors, and initial risk assignment will require pathology and
surgical rapid central reviews. The Specimen Transmittal Form and Pre
Treatment Pathology Checklist are also needed.
• Please note: if the above required items are not received within 120 days of
study enrollment, the patient will be considered off study
• 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
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children (BEGIN)
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly
effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their
impact on children with cystic fibrosis (CF).
Christina Barreda
All
up to 5 Years old
NA
This study is NOT accepting healthy volunteers
NCT04509050
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Inclusion Criteria:
• Part A:
• Less than 5 years of age at the first study visit.
• Documentation of a CF diagnosis.
Part B:
• Participated in Part A OR less than 6 years of age at the first study visit.
• Documentation of a CF diagnosis.
• CFTR mutations consistent with FDA labeled indication of highly effective modulator
therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor).
• Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.
Exclusion Criteria:
• Part A and Part B:
Use of an investigational drug within 28 days prior to and including the first study visit.
Use of ivacaftor or elexacaftor/tezacaftor/ivacaftor within the 180 days prior to and
including the first study visit.
Use of chronic oral corticosteroids within the 28 days prior to and including the first
study visit.
Cystic Fibrosis, Cystic fibrosis, Other, Children's & Adolescent Health
The primary purpose of this study is to discover new disease genes for rare Mendelian
disorders and its secondary purpose include diagnosing people with rare genetic disorders
that have not been previously diagnosed through conventional clinical means, learning more
about the pathobiology of genetic disorders, and developing novel diagnostic technologies and
analytics. 500 participants with undiagnosed and suspected genetic disorders will be
recruited over approximately 5 years time.
Stephen Meyn
All
up to 100 Years old
NA
This study is NOT accepting healthy volunteers
NCT04586075
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Inclusion Criteria:
• The applicant has a condition that remains undiagnosed despite thorough evaluation by
healthcare providers (including clinical genetic testing).
• The applicant has at least one objective finding that is likely to have an
identifiable genetic etiology.
• The applicant likely has a currently undescribed/new genetic condition or a known
genetic condition associated with a novel gene.
• The applicant/legal guardian agrees to the collection, storage and recurrent sharing
of coded information and biomaterials for research and diagnostic purposes both within
and outside of the University of Wisconsin-Undiagnosed Diseases Program (UW-UDP)
• The applicant/legal guardian agrees to receive secondary findings from genetic
testing.
• The applicant/legal guardian has sufficient proficiency in English to understand the
consent.
Exclusion Criteria:
• The applicant already has a diagnosis that explains the objective findings.
• A specific diagnosis is suspected and a standard clinical workup performed by the
referring/primary care provider would be appropriate.
• The UW-UDP is unlikely to improve on the comprehensive workup the applicant has
already received.
• The applicant's symptoms are likely multifactorial or due to a non-genetic cause.
Rare Diseases, Genetic Disease, Undiagnosed Disease, Other specified congenital malformation syndromes affecting multiple systems, Other
Inotuzumab Ozogamicin and Blinatumomab in Treating Patients With Newly Diagnosed, Recurrent, or Refractory CD22-Positive B-Lineage Acute Lymphoblastic Leukemia
This phase II trial studies how well inotuzumab ozogamicin and blinatumomab work in treating
patients with CD22-positive B-lineage acute lymphoblastic leukemia that is newly diagnosed,
has come back, or does not respond to treatment. Immunotherapy with monoclonal antibodies,
such as inotuzumab ozogamicin and blinatumomab, may help the body's immune system attack the
cancer, and may interfere with the ability of tumor cells to grow and spread.
Ryan Mattison, MD
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03739814
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Inclusion Criteria:
• Pre-registration Eligibility Criteria (Step 0)
• Submission of bone marrow aspirate and peripheral blood for MRD analysis is mandatory
prior to registration; the bone marrow sample should be from the first aspiration
(i.e. first pull). Aspirate needle should be redirected if needed to get first pull
bone marrow aspirate. It should be initiated as soon as possible after
pre-registration. The specimens should be sent to the HEME Biobank.
• Lumbar Puncture (Spinal Tap) and Intrathecal Methotrexate:
• Patients may receive the day 1 of course IA dose of intrathecal (IT)
methotrexate during the prior-to-registration lumbar puncture (or the venous
line placement) to avoid a second lumbar puncture. If the dose is
administered prior to registration, then systemic chemotherapy must begin
within 7 days of this IT chemotherapy.
• Registration Eligibility Criteria (Step 1)
• Morphologic diagnosis of precursor B-cell acute lymphoblastic leukemia (ALL) based on
World Health Organization (WHO) criteria. Patients with Burkitt lymphoma/leukemia are
not eligible.
• CD22-positive disease defined as CD22 expression by >= 20% of lymphoblasts by local
hematopathology evaluation.
• Philadelphia chromosome/BCR-ABL1-negative ALL by cytogenetics, fluorescence in situ
hybridization (FISH), and/or polymerase chain reaction (PCR). If any test is positive
for Philadelphia chromosome/BCR-ABL1, then the patient is ineligible.
• No active central nervous system (CNS) leukemia (i.e. only CNS-1 disease allowed).
Active CNS leukemia is defined as morphologic evidence of lymphoblasts in the
cerebrospinal fluid (CSF), use of CNS-directed local treatment for active disease
within 28 days prior to registration, symptomatic CNS leukemia (i.e. cranial nerve
palsies or other significant neurological dysfunction) within the 28 days prior to
registration, and/or known asymptomatic parenchymal CNS mass lesions; see below for
additional guidance. Prophylactic intrathecal medication alone is not an exclusion.
• Categories of CNS Involvement for CNS Evaluation Prior to Registration:
• CNS 1: CSF has < 5 WBC/uL with cytospin negative for blasts; or >= 10 red
blood cell (RBC)/uL with cytospin negative for blasts.
• CNS 2: CSF has < 5 WBC/uL with cytospin positive for blasts; or >= 10 RBC/uL
with cytospin positive for blasts; or >= 10 RBC/uL, WBC/uL >= 5 but less
than Steinherz/Bleyer algorithm with cytospin positive for blasts (see
below).
• CNS 3: CSF has >= 5 WBC/uL with cytospin positive for blasts; or >= 10
RBC/uL, >= 5 WBC/uL and positive by Steinherz/Bleyer algorithm (see below);
or clinical signs of CNS leukemia (such as facial nerve palsy, brain/eye
involvement or hypothalamic syndrome). Steinherz/Bleyer Method of Evaluating
Initial Traumatic Lumbar Punctures:
• If the patient has leukemia cells in the peripheral blood and the
lumbar puncture is traumatic and contains >= 5 WBC/uL with blasts, the
following algorithm should be used to define CNS disease: CSF WBC/CSF
RBC > 2 x (Blood WBC/Blood RBC count)
• Patients with known or suspected testicular involvement by leukemia are allowed
provided that the patient receives concomitant scrotal/testicular radiotherapy.
• Unilateral or bilateral testicular enlargement should be assessed by ultrasound
or other imaging technique. Biopsy is recommended if clinical findings are
equivocal or suggestive of hydrocele or a non-leukemic mass, but further
assessments are per treating physician discretion.
• Not pregnant and not nursing.
• This study involves agents that have 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.
• Eastern Cooperative Oncology Group (ECOG) performance status: 0-2
• No unstable cardiac disease such as myocardial infarction, angina pectoris,
uncontrolled heart failure, or uncontrolled cardiac arrhythmia within 6 months of
registration.
• No impaired cardiac function, defined as left ventricular ejection fraction (LVEF) <
45% or New York Heart Association (NYHA) stage III or IV congestive heart failure
(CHF).
• Patients with known human immunodeficiency virus (HIV) infection are eligible if they
have been on effective antiretroviral therapy with an undetectable viral load tested
within 6 months of registration.
• Patients with hepatitis B virus (HBV) are eligible only if they meet all the
following:
• On HBV-suppressive therapy.
• No evidence of active virus.
• No evidence of HBV-related liver damage.
• Patients with hepatitis C virus (HCV) are eligible only if they meet all the
following:
• Successfully completed complete-eradication therapy with undetectable viral load.
• No evidence of HCV-related liver damage.
• No history of clinically relevant neurologic disorder such as epilepsy, seizure,
aphasia, stroke, severe brain injury, structural brain abnormality, benign brain
tumor, dementia, Parkinson's disease, movement disorder, cerebellar disease, or other
significant CNS abnormalities.
• No prior additional malignancy (i.e. in addition to ALL) except adequately treated
basal- or squamous-cell skin cancer, in situ cervical cancer, stage I or II cancer
from which the patient is currently in complete remission, or any other cancer from
which the patient has been disease-free for >= 2 years.
• No history of clinically significant ventricular arrhythmia, unexplained non-vasovagal
syncope, or chronic bradycardic states such as sinoatrial block or higher degree of
atrioventricular block unless a permanent pacemaker has been implanted.
• No history of chronic liver disease, including cirrhosis.
• No history of sinusoidal occlusion syndrome/veno-occlusive disease of the liver.
• No uncontrolled infection or recent history (within 4 months prior to registration) of
deep tissue infections such as fasciitis or osteomyelitis.
• Total bilirubin, serum =< 1.5 x upper limit of normal (ULN)*
• Except in the event of: 1) Gilbert disease, in which case total bilirubin must be
=< 2 x ULN, or 2) elevated bilirubin believed by investigator to be due to
leukemic infiltration, in which case total bilirubin must be =< 2 x ULN.
• Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) =< 2.5 x ULN
• Creatinine, serum =< 1.5 ULN OR creatinine clearance >= 40 mL/min
• QT interval by Fridericia's correction formula (QTcF) =< 470 msec
• Cohort 1 Patients Only
• Age >= 60 years.
• No prior treatment for ALL except a single dose of intrathecal chemotherapy,
corticosteroids, hydroxyurea, and/or leukapheresis to reduce peripheral blast count
and prevent ALL complications. Allowed therapy may be administered for no more than 14
days and must be completed >= 24 hours prior to the initiation of protocol therapy.
• No plan for allogeneic or autologous hematopoietic cell transplantation (HCT).
• Cohort 2 Patients Only:
• Age >= 18 years.
• Relapsed or refractory disease in salvage 1 or 2.
• No isolated extramedullary relapse.
• Prior allogeneic HCT permitted.
• Patients with prior allogeneic HCT must have completed transplantation >= 4 months
prior to registration.
• Patients with prior allogeneic HCT must have no evidence of graft-versus-host disease
and must have completed immunosuppressive therapy >= 30 days prior to registration.
• Prior treatment with inotuzumab ozogamicin, blinatumomab, other CD22-directed therapy,
or other CD19-directed therapy is not allowed.
• Prior treatment with rituximab must be completed >= 7 days prior to registration.
• Prior treatment with other monoclonal antibodies must be completed >= 6 weeks prior to
registration.
• Prior treatment for ALL must be completed >= 14 days prior to registration with the
following exceptions: intrathecal chemotherapy, hydroxyurea, corticosteroids,
6-mercaptopurine, methotrexate, vincristine, and/or leukapheresis to reduce
circulating absolute lymphoblast count to =< 10,000/uL or prevent complications
related to ALL are allowed but must be completed >= 24 hours prior to the initiation
of protocol therapy.
• Patients should have resolution of any acute non-hematologic toxicities of prior
therapy to National Cancer Institute (NCI) Common Terminology Criteria for Adverse
Events (CTCAE) version (v)5.0 grade =< 1.
• Peripheral blood absolute lymphoblast count =< 10,000/uL (treatment allowed as above
to reduce blast count to =< 10,000/uL)
B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative, Recurrent B Acute Lymphoblastic Leukemia, Refractory B Acute Lymphoblastic Leukemia, Lymphoid Leukemia, Leukemia
Preparing for Prevention of Huntington's Disease (PREVENT-HD) (PREVENT-HD)
This is a prospective investigation which aims to address key challenges to the design of
clinical trials to prevent the onset of Huntington's disease (HD). The project will provide
necessary psychometric data for clinical outcome assessments (COAs) and biomarkers (BMs) in
the cerebral spinal fluid (CSF) to address questions of central importance to the success of
these measures for premanifest clinical trials. Of the 258 participants: 52 will be low risk
of motor diagnosis, 102 high risk of motor diagnosis, 52 with diagnosed HD (stages I or II),
and 52 healthy controls. Participants can expect to be in the study for up to 2 years.
Jane Paulsen
All
18 Years to 80 Years old
NA
This study is also accepting healthy volunteers
NCT04818060
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Inclusion Criteria for HD Participants:
• Estimated at low or high probability of motor diagnosis based on the multivariate risk
score (MRS)
• Willing to commit to two in-person assessment visits (baseline and 2-year follow-up)
and remote assessments as needed
• No active comorbidities (i.e. receiving stable treatment)
• All medications will be allowed although the protocol will mandate documentation of
medications and analyses will particularly assess potential impact of medications on
outcomes (i.e., sedation of abnormal movements)
• If previously measured, CAG results must be 36 or above (previous CAG is not a
requirement, but this threshold will be upheld for those participants who have their
CAG scores and/or have them in their medical record).
Inclusion Criteria for Healthy Controls (HC):
• Willing to commit to two in-person assessment visits (baseline and 2-year follow-up)
and remote assessments as needed
• In generally good health
• IQ > 70
• Able to undergo an MRI scan
Exclusion Criteria (for all Participants):
• Evidence of unstable medical or psychiatric illness (including substance abuse)
• History of severe learning disability, mental retardation, or other central nervous
system (CNS) disease or event (e.g., seizures, head trauma, additional neurological
diagnoses)
• Treatment with phenothiazine-derivative antiemetic medications such as
prochlorperazine, metoclopramide, promethazine and Inapsine greater than 3 times per
month
• History of serious alcohol or drug abuse within the past year
• Unable (determined by patient's prescribing doctor) to not take tryptophan, leucine,
niacin or niacinamide-containing dietary supplements, anti-inflammatory medications,
anti-coagulants (such as warfarin and heparin) or anti-platelets (such as aspirin) in
the past 14 days to assure safety during lumbar puncture
• Unable to fast (no food or drink, only water) overnight before the lumbar puncture
Prospective Study of Pregnancy in Women With Cystic Fibrosis (MAYFLOWERS)
In this study, the investigators aim to evaluate changes in lung function in women with
cystic fibrosis (CF) during pregnancy and for 2 years after pregnancy based on exposure to
highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators.
Erin Lowery
Female
16 Years and over
NA
This study is NOT accepting healthy volunteers
NCT04828382
Show full eligibility criteria
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Inclusion Criteria:
• Pregnant, intending to continue pregnancy, enrolled in the Cystic Fibrosis Foundation
Patient Registry (CFFPR)
Exclusion Criteria:
• None
Pregnancy Related, Cystic Fibrosis, Cystic fibrosis, Other
Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (EPIK-P2)
This is a prospective Phase II multi-center study with an upfront 16-week, randomized,
double-blind, placebo-controlled period, and extension periods, to assess the efficacy,
safety and pharmacokinetics of alpelisib in pediatric and adult participants with
PIK3CA-related overgrowth spectrum (PROS).
Beth Drolet, MD
All
2 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04589650
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Inclusion Criteria:
1. Signed informed consent and assent (when applicable) from the patient, parent, legal
authorized representative or guardian prior to any study related screening procedures
are performed
2. Patients with diagnosis of PROS with symptomatic and /or progressive overgrowth and at
least one measurable PROS-related lesion confirmed by blinded independent review
committee (BIRC) assessment
3. Documented evidence of a somatic mutation(s) in the PIK3CA gene performed in local
laboratories
4. A tissue sample (fresh or archival) is be sent to a Novartis-designated central
laboratory. If archival tissue is not available, collection of a fresh tissue biopsy
is required for participants in Groups 1, 2 and 5, if it is not clinically
contraindicated. For participants in Groups 3 and 4, a fresh tissue biopsy is not
mandatory.
For China only: Tissue sample collection and biomarker assessments are not applicable.
For Germany only: If archival tissue is available, it must be sent to a Novartis
designated central laboratory. If no archival tissue is available, obtaining a fresh
tissue biopsy is recommended, if it is not clinically contraindicated, but is not
mandatory.
5. Karnofsky (in patients > 16 years old at study entry)/Lansky (≤16 yrs of age at study
entry) performance status index ≥50
6. Adequate bone marrow and organ function including Fasting plasma glucose (FPG) ≤ 140
mg/dL (7.7 mmol/L) and Glycosylated hemoglobin (HbA1c) ≤ 6.5% (both criteria have to
be met) (as assessed by central laboratory for eligibility)
7. Presence of at least one PROS-related measurable lesion defined as a lesion with
longest diameter ≥2 cm, when the volume can be accurately and reproducibly measured by
MRI (Magnetic resonance imaging), and associated with complaints, clinical symptoms or
functional limitations affecting the patient's everyday life. Measurability must be
confirmed by BIRC before randomization.
Exclusion Criteria:
1. Participant with only isolated macrodactyly, skin nevus/nevi and macroencephaly (the
only clinical feature or a combination of any of three of them), in absence of other
PROS-related lesions at the time of informed consent
2. Previous treatment with alpelisib and/or any other PI3K inhibitor(s) (except treatment
attempt, defined as the attempt to treat PROS with any of PI3K inhibitors, with
treatment duration less than 2 weeks and stopped at least 4 weeks prior to the first
dose of study medication with alpelisib)
3. Radiation exposure for PROS treatment purpose within the previous 12 months on those
PROS areas which are expected to qualify for target lesions (except lesion(s)
progressing after completion of radiotherapy) at time of informed consent.
4. Debulking or other major surgery performed within 3 months at time of informed consent
5. Clinically meaningful PROS-related thrombotic event (Grade 2 and more as per CTCAE
v.4.03) within 30 days before informed consent, and/or sclerotherapy/embolization for
vascular complications performed within 6 weeks before informed consent. Note:
Participants receiving anticoagulants for PROS-related coagulopathy, primary or
secondary prophylaxis of thrombosis may be included in the study
6. Participants in Groups 1, 2 ad 5 with documented pneumonitis or interstitial lung
disease at time of informed consent and with impaired lung function (e.g., FEV1 or
DLCO ≤ 70% of predicted) that is not related to PROS. Participants in Groups 3 and 4
with documented or suspicious pneumonitis or interstitial lung disease based on MRI
images at time of informed consent
7. History of acute pancreatitis within 1 year before informed consent or past medical
history of chronic pancreatitis at time of informed consent
8. Participants with an established diagnosis of type I diabetes mellitus or uncontrolled
type II diabetes mellitus at time of informed consent
9. Known history of seizure, or epilepsy, regardless of relatedness to PROS spectrum at
time of informed consent, when epilepsy is not controlled and/or the patient may not
be switched to non-enzyme inducing antiepileptic drug(s) at time of informed consent.
10. Participants with clinically significant worsening of PROS-related laboratory
anomalies, physical signs and symptoms (such as, but not limited to increase of
D-dimers, worsening of underlying pain, newly occurring swelling or redness)
indicating an uncontrolled condition during the screening phase, particularly if
systemic treatment with any other inhibitor of the PI3K/AKT/mTOR pathway was stopped
prior to the start of study treatment. This includes but is not limited to
hypercoagulability state in participants not receiving prophylactic treatment.
Other inclusion/exclusion criteria may apply
The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study
This study aims to use clinical and biological characteristics of acute leukemias to screen
for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and
blood from patients with leukemia that has come back after treatment or is difficult to treat
may provide information about the patient's leukemia that is important when deciding how to
best treat it, and may help doctors find better ways to diagnose and treat leukemia in
children, adolescents, and young adults.
Kenneth Desantes, M.D.
All
up to 22 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04726241
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Inclusion Criteria:
• Patients must be less than 22 years of age at the time of study enrollment
• Patient must have one of the following:
• Patient has known or suspected relapsed/refractory (including primary refractory)
AML
• This includes isolated myeloid sarcoma
• Patient has known or suspected relapsed/refractory (including primary refractory)
myeloid leukemia of Down syndrome
• Patient has known or suspected relapsed ALL that meets one of the following
criteria:
• Second or greater B-ALL medullary relapse, excluding KMT2Ar.
• Any first or greater B-ALL medullary relapse involving KMT2Ar.
• Any first or greater T-ALL medullary relapse with or without KMT2Ar.
• Patient has known or suspected relapsed/refractory (including primary refractory)
mixed phenotype acute leukemia (MPAL)
• Patient has known or suspected de novo or relapsed/refractory (including primary
refractory) treatment-related AML (t-AML) or treatment-related myelodysplastic
syndrome (t-MDS)
• Patient has known or suspected de novo or relapsed/refractory (including primary
refractory) myelodysplastic syndrome (MDS)
• Patient has known or suspected de novo or relapsed/refractory (including primary
refractory) juvenile myelomonocytic leukemia (JMML)
• 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
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Juvenile Myelomonocytic Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome Post Cytotoxic Therapy, Myeloid Leukemia Associated With Down Syndrome, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Leukemia, other, Leukemia
A phase 3 randomized partial blind storage duration ranging study in patients undergoing
complex cardiac surgery that will compare the transfusion of cold stored platelets to
standard room temperature stored platelets. The primary objective is to establish that cold
stored platelets have a non-inferiority (or superiority) to room temperature platelets.
Eric Simon
All
29 Days to 84 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04834414
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Inclusion Criteria:
• Age greater than 28 days and less than 85 years
• Planned complex cardiac surgery with planned use of cardiopulmonary bypass
Exclusion Criteria:
• Expected order for washed or volume reduced platelets
• Patient with known anti-platelet antibodies
• Platelet transfusion refractoriness due to anti-HLA antibodies
• Known or suspected pregnancy
• Previously randomized in this study
• Conscious objection or unwillingness to receive blood products
• Known IgA deficiency
• Known congenital platelet disorder
• Known congenital bleeding disorder
• Planned post-operative extracorporeal membrane oxygenation (ECMO), ventricular assist
device (VAD), and/or continuous renal replacement therapy (CRRT)/ hemodialysis
• Patients intended to receive whole blood either intra-operative or post-operative for
bleeding
• Platelet transfusion (of any type) within 24 hours prior to the date of surgery
• Pre-operative thrombocytopenia, defined as platelet count <75x10(9)/L, based on the
most recent labs completed within 72 hours prior to the date of surgery.
Acute Blood Loss, Complications of heart transplant, Congenital malformation of heart, unspecified, Heart failure, Other acute ischemic heart diseases, Other, Heart & Vascular
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
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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 Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of
VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
Andrew Braun
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05444257
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Key
Inclusion Criteria:
• Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and
VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but
did not permanently discontinue study drug, and completed study visits up to the last
scheduled visit of the Treatment Period in the parent study
Key
Exclusion Criteria:
• History of drug intolerance in a parent study
• Pregnant or breast-feeding females
Other protocol defined Inclusion/Exclusion criteria may apply.
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and
efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at
least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane
conductance regulator (CFTR) gene.
Hara Levy, MD
All
1 Year to 11 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05422222
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Key
Inclusion Criteria:
• Participants with stable CF and at least 1 TCR mutation (including F508del) in the
CFTR gene
Key
Exclusion Criteria:
• History of solid organ, hematological transplantation, or cancer
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh
Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
• Lung infection with organisms associated with a more rapid decline in pulmonary status
Other protocol defined Inclusion/Exclusion criteria may apply.
Standardizing Treatments for Pulmonary Exacerbations - Aminoglycoside Study (STOP360AG)
The purpose of this study is to look at pulmonary exacerbations in people with cystic
fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a
vein (intravenous or IV). A pulmonary exacerbation is a worsening of respiratory symptoms in
people with CF that needs medical intervention. Both doctors and CF patients are trying to
understand the best way to treat pulmonary exacerbations. This study is trying to answer the
following questions about treating a pulmonary exacerbation:
- Do participants have the same improvement in lung function and symptoms if they are
treated with one type of antibiotic (called beta-lactams or β-lactams) versus taking two
different types of antibiotics (tobramycin and β-lactams)?
- Is taking one type of antibiotic just as good as taking two types?
Andrew Braun
All
6 Years and over
Phase 4
This study is NOT accepting healthy volunteers
NCT05548283
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Inclusion Criteria:
• All genders ≥ 6 years of age at Visit 1
• Documentation of a CF diagnosis
• Clinician intent to treat index CF PEx with a planned 14-day course of IV
antimicrobials
• At least one documented Pa positive culture within two years prior to Visit 1
Exclusion Criteria:
• Participant is not pregnant
• No known renal impairment or history of solid organ transplantation
• No IV antimicrobial treatment, ICU admission, pneumothorax, or hemoptysis within 6
weeks prior to Visit 1
• No use of investigational therapies, new CF transmembrane conductance regulator (CFTR)
modulators, or treatment for Nontuberculous mycobacteria (NTM) within 4 weeks prior to
Visit 1
• No history of hypersensitivity, vestibular, or auditory toxicity with aminoglycosides
• No more than one day of IV aminoglycosides administered for the current PEx treatment
prior to Visit 1
Cystic Fibrosis, Cystic Fibrosis Pulmonary Exacerbation, Other
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
The purpose of this study is to evaluate the safety, and tolerability and efficacy of VX-522
in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis
transmembrane conductance regulator (CFTR) genotype not responsive to CFTR modulator therapy.
Andrew Braun
All
18 Years to 65 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT05668741
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Key
Inclusion Criteria:
• Body mass index is less than (<) 30.0 kilograms per meter square (kg/m^2)
• A total body weight greater than (>) 50 kg
• Stable CF disease
• CFTR gene mutations on both alleles that are not responsive to CFTR modulator therapy
o Example mutations include but are not limited to, mutations that do not produce CFTR
protein (i.e., Class I): nonsense mutations (e.g., G542X, W1282X) and canonical splice
mutations (e.g., 621+1G->T)
• Forced expiratory volume in 1 second (FEV1) value for SAD: greater than or equal to
(≥)40 percent (%), MAD: ≥ 40% to less than or equal to (≤) 90%
Key
Exclusion Criteria:
• History of uncontrolled asthma within a year prior to screening
• History of solid organ or hematological transplantation
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh
Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
• Arterial oxygen saturation on room air less than (<) 94% at screening
Other protocol defined Inclusion/Exclusion criteria may apply.
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of
vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis
(CF).
Hara Levy, MD
All
1 Year and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05844449
Show full eligibility criteria
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Key
Inclusion Criteria:
• Participants who have completed study drug treatment in the parent study
(VX21-121-105; NCT Number: NCT05422222)
Key
Exclusion Criteria:
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe
hepatic impairment that might pose an additional risk in administering study drug
• History of solid organ, hematological transplantation, or cancer
• History of drug intolerance in the parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
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