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ClinicalTrials.gov public records Last synced Jul 22, 2026, 11:50 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Adrenocortical Carcinoma, Gastrointestinal Carcinoid Tumor, Kidney Cancer, Neuroblastoma, Retinoblastoma, Sarcoma
Interventions
Seneca Valley virus-001, cyclophosphamide, laboratory biomarker analysis, pharmacological study
Biological · Drug · Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
3 Years to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
18
States / cities
Birmingham, Alabama • Orange, California • San Francisco, California + 15 more
Conditions
Beta-Thalassemia
Interventions
arginine butyrate, epoetin alfa
Drug
Lead sponsor
Boston University
Other
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
6
States / cities
Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more
Conditions
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent
Interventions
Myeloablative Chemotherapy, Stem Cell Rescue
Procedure
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2010
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
AAV directed hLDLR gene therapy
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
5
States / cities
Boca Raton, Florida • Kansas City, Kansas • Portland, Oregon + 2 more
Conditions
Kartagener Syndrome, Cystic Fibrosis, Pseudohypoaldosteronism, Primary Ciliary Dyskinesia
Interventions
Not listed
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
367 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
7
States / cities
Palo Alto, California • Denver, Colorado • Bethesda, Maryland + 3 more
Conditions
Turner's Syndrome
Interventions
Estrogen
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 12 Years · Female only
Enrollment
260 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1990
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Completed Not applicable Interventional

Study of NTBC for Tyrosinemia I

NCT00004443
Conditions
Tyrosinemia I
Interventions
NTBC
Drug
Lead sponsor
University of Washington
Other
Eligibility
Up to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2006
U.S. locations
2
States / cities
Portland, Oregon • Seattle, Washington
Conditions
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Interventions
Non-investigational
Other
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
8
States / cities
Torrance, California • Denver, Colorado • Washington D.C., District of Columbia + 5 more
Recruiting No phase listed Observational Accepts healthy volunteers

Vascular Disease Discovery Protocol

NCT03538639
Conditions
Vascular Dysfunction, Genetic Mutations, Genetic Predisposition
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Genetic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2028
U.S. locations
33
States / cities
Birmingham, Alabama • Huntsville, Alabama • Los Angeles, California + 21 more
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Conditions
Huntington Disease
Interventions
PTC518, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
25 Years and older
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
4
States / cities
La Jolla, California • Nashville, Tennessee • Houston, Texas + 1 more
Conditions
Osteogenesis Imperfecta
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2031
U.S. locations
11
States / cities
Phoenix, Arizona • Los Angeles, California • Wilmington, Delaware + 8 more
Conditions
Hyperaldosteronism
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
71
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 59 more
Conditions
Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease, Development, Infant, Development, Child
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 4 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
AAVrh.10CUCLN2
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
New York, New York
Enrolling by invitation Not applicable Interventional

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH Assessment
Diagnostic Test
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
22 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 1 more
Conditions
Homozygous Familial Hypercholesterolemia (HoFH)
Interventions
Not listed
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boca Raton, Florida
Conditions
Cystinosis
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
La Jolla, California
Conditions
Mondini Dysplasia
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Ann Arbor, Michigan
Conditions
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue, Clear Cell Sarcoma of Soft Tissue, Ewing Sarcoma, Hepatoblastoma, Hepatocellular Carcinoma, Osteosarcoma, Recurrent Adrenal Cortical Carcinoma, Recurrent Alveolar Soft Part Sarcoma, Recurrent Clear Cell Sarcoma of Soft Tissue, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Kidney Wilms Tumor, Recurrent Malignant Solid Neoplasm, Recurrent Osteosarcoma, Recurrent Primary Malignant Central Nervous System Neoplasm, Recurrent Renal Cell Carcinoma, Recurrent Rhabdomyosarcoma, Recurrent Soft Tissue Sarcoma, Recurrent Thyroid Gland Medullary Carcinoma, Refractory Adrenal Cortical Carcinoma, Refractory Alveolar Soft Part Sarcoma, Refractory Clear Cell Sarcoma of Soft Tissue, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Osteosarcoma, Refractory Primary Central Nervous System Neoplasm, Refractory Primary Malignant Central Nervous System Neoplasm, Refractory Renal Cell Carcinoma, Refractory Rhabdomyosarcoma, Refractory Soft Tissue Sarcoma, Refractory Thyroid Gland Medullary Carcinoma, Refractory Wilms Tumor, Renal Cell Carcinoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Solid Neoplasm, Thyroid Gland Medullary Carcinoma, Wilms Tumor
Interventions
Cabozantinib, Cabozantinib S-malate, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 30 Years
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
142
States / cities
Birmingham, Alabama • Anchorage, Alaska • Little Rock, Arkansas + 104 more
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more