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Conditions: Lymphoma, Non-Hodgkin, Multiple Myeloma, Advanced Solid Tumors
Interventions: Sunitinib, Temsirolimus, Trastuzumab and Pertuzumab, Vemurafenib and Cobimetinib, Olaparib, Nivolumab and Ipilimumab, Abemaciclib, Talazoparib, Atezolizumab and Talazoparib, Tucatinib plus Trastuzumab Subcutaneous (SC), Futibatinib, Dabrafenib plus Trametinib, Fam-Trastuzumab Deruxtecan-Nxki (TDxD); Drug
Lead sponsor: American Society of Clinical Oncology; Other
Eligibility: 12 Years and older
Enrollment: 4,200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 174
States / cities: Birmingham, Alabama • Phoenix, Arizona • Auburn, California + 137 more

Conditions: NF type1 With Inoperable Plexiform Neurofibromas
Interventions: Selumetinib; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 2 Years to 130 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: New Orleans, Louisiana

Conditions: Friedreich's Ataxia
Interventions: Not listed
Lead sponsor: University of Florida; Other
Eligibility: 6 Years to 70 Years
Enrollment: 203 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2030
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease
Interventions: Campath, Sirolimus, Base-edited hematopoietic stem and progenitor cells, Busulfan, Palifermin, Filgrastim, Plerixafor; Drug · Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease
Interventions: Inclacumab, Placebo; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years and older
Enrollment: 72 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 17
States / cities: Mobile, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 11 more

Conditions: X-Linked Retinitis Pigmentosa
Interventions: rAAV2tYF-GRK1-RPGR; Biological
Lead sponsor: Beacon Therapeutics; Industry
Eligibility: 8 Years to 50 Years · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 6
States / cities: Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more

Conditions: Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Interventions: Cobimetinib, Enasidenib Mesylate; Drug
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Duarte, California

Conditions: Arrhythmogenic Right Ventricular Cardiomyopathy
Interventions: Not listed
Lead sponsor: Tenaya Therapeutics; Industry
Eligibility: 14 Years to 65 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 8
States / cities: San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 5 more

Conditions: Human Immunodeficiency Virus (HIV)
Interventions: ZFN Modified CD4+ T Cells, Cyclophosphamide; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Breast Neoplasms
Interventions: Dexamethasone, Trabectedin; Drug
Lead sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.; Industry
Eligibility: 18 Years and older
Enrollment: 127 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 21
States / cities: Sedona, Arizona • Denver, Colorado • Torrington, Connecticut + 18 more

Conditions: Breast Cancer, Breast Cancer Stage IV, HER2-negative Breast Cancer, Solid Carcinoma, MEK1 Gene Mutation, MEK2 Gene Mutation, Metastatic Breast Cancer
Interventions: Mirdametinib, Fulvestrant; Drug
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 4
States / cities: Middletown, New Jersey • Commack, New York • Harrison, New York + 1 more

Conditions: Breast Cancer, ER-positive Breast Cancer, HER2-negative Breast Cancer, Metastatic Breast Cancer
Interventions: AI+CDK4/6i, SERD+CDK4/6i, mTOR inhibitor + AI, mTOR inhibitor + SERD, mTOR inhibitor + Selective estrogen receptor modulator, PI3K inhibitor + SERD, PI3K inhibitor + AI, Chemotherapy, Oral SERD, PARPi, AKT inhibitor, Step 3 Arm 2; Drug · Other
Lead sponsor: University of Miami; Other
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Ovarian Cancer, Primary Peritoneal Cancer, Serous Carcinoma Cancer, Triple-Negative Breast Cancer, Fallopian Tube Cancer
Interventions: ABT-888, Cyclophosphamide; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 124 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 9
States / cities: Davis, California • Tampa, Florida • Chicago, Illinois + 6 more

Conditions: PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome
Interventions: RAD001, Placebo; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 5 Years to 45 Years
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 3
States / cities: Palo Alto, California • Boston, Massachusetts • Cleveland, Ohio

Conditions: Acute Myeloid Leukemia
Interventions: Decitabine, Nivolumab, Venetoclax; Drug · Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Dystrophic Epidermolysis Bullosa
Interventions: PTW-002 10 mg/g gel, Placebo; Drug
Lead sponsor: Phoenicis Therapeutics; Industry
Eligibility: 4 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 3
States / cities: Stanford, California • Worcester, Massachusetts • Cincinnati, Ohio

Conditions: BRCA1 Mutation Carrier, BRCA2 Mutation Carrier, Breast Carcinoma
Interventions: Laboratory Biomarker Analysis; Other
Lead sponsor: Gynecologic Oncology Group; Network
Eligibility: 18 Years to 80 Years · Female only
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2008
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Platinum Sensitive Relapsed Ovarian Cancer
Interventions: OLAPARIB, PLACEBO; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 96 Years · Female only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 15
States / cities: Mobile, Alabama • Phoenix, Arizona • Los Angeles, California + 12 more

Conditions: Myotubular Myopathy
Interventions: Genetic Testing; Other
Lead sponsor: Cure CMD; Other
Eligibility: 30 Days and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2017
U.S. locations: 1
States / cities: Torrance, California

Conditions: Li-Fraumeni Syndrome, TP53 Gene Mutation, Hereditary Cancer Syndrome, Clonal Hematopoiesis, Mosaicism
Interventions: Data and Specimen Collection; Genetic
Lead sponsor: Dana-Farber Cancer Institute; Other
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2032
U.S. locations: 3
States / cities: Boston, Massachusetts

Conditions: Metastatic HPV-16 Positive Squamous Cell Anal Cancer
Interventions: Cyclophosphamide, Fludarabine, Aldesleukin, Pembrolizumab (KEYTRUDA(R)), Individual Patient TCR-Transduced PBL; Drug · Biological
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years to 70 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Low Grade Glioma, Recurrent Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma, Recurrent Hepatoblastoma, Recurrent Hodgkin Lymphoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Germ Cell Tumor, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Medulloblastoma, Recurrent Neuroblastoma, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Recurrent Peripheral Primitive Neuroectodermal Tumor, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Refractory Hodgkin Lymphoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Germ Cell Tumor, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Refractory Peripheral Primitive Neuroectodermal Tumor, Refractory Rhabdoid Tumor, Refractory Soft Tissue Sarcoma, Rhabdoid Tumor, Stage III Soft Tissue Sarcoma AJCC v7, Stage IV Soft Tissue Sarcoma AJCC v7, Wilms Tumor
Interventions: Tazemetostat; Drug
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 12 Months to 21 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 115
States / cities: Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more

Conditions: Fabry Disease
Interventions: Not listed
Lead sponsor: Baylor Research Institute; Other
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 107 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2015
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: BAG3 Mutation Associated Dilated Cardiomyopathy
Interventions: ALXN2350; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 7
States / cities: Birmingham, Alabama • Palo Alto, California • Boston, Massachusetts + 4 more

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TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1

Biomarkers in Friedreich's Ataxia

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

A Phase I Study of T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728mR in HIV-Infected Patients

An Efficacy Study of Trabectedin in the Treatment of Participants With Specific Subtypes of Metastatic Breast Cancer

A Study of Mirdametinib on Its Own or in Combination With Fulvestrant in People With Solid Tumor Cancer

Levels of Circulating Tumor DNA as a Predictive Marker for Early Switch in Treatment for Patients With Metastatic (Stage IV) Breast Cancer

Phase II ABT-888 With Cyclophosphamide

RAD001 and Neurocognition in PTEN Hamartoma Tumor Syndrome

Testing Nivolumab in Combination With Decitabine and Venetoclax in Patients With Newly Diagnosed TP53 Gene Mutated Acute Myeloid Leukemia

Study of PTW-002 in Patients With Dominant or Recessive Dystrophic Epidermolysis Bullosa Due to Mutation(s) in Exon 73 of the COL7A1 Gene

Breast Cancer Risk in Women Who Are BRCA1/BRCA2 Mutation Carriers

Olaparib Maintenance Treatment Versus Placebo in Patients With PSR Ovarian Cancer Who Are in CR or PR to Platinum-based Chemotherapy and Whose Tumours Carry sBRCAm or HRR-associated Genes Mutations

Myotubular Myopathy Genetic Testing Study

Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress

Administration of Autologous T-Cells Genetically Engineered to Express T-Cell Receptors Reactive Against Mutated or Viral Neoantigens in a Patient With Metastatic Cancer Plus the Administration of Pembrolizumab

Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)

Androgenetic Alopecia in Fabry Disease

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy