Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 29, 2026, 9:47 AM EDT

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Showing 1–24 of 910 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
CAP-1002, Placebo
Biological · Drug
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Orlando, Florida + 6 more
Conditions
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
Interventions
HB-adMSCs
Biological
Lead sponsor
Hope Biosciences Research Foundation
Industry
Eligibility
Not listed
U.S. locations
1
States / cities
Sugar Land, Texas
Conditions
Cerebral Palsy, Muscle Disorder, Physical Disability, Posture Disorders in Children, Spina Bifida
Interventions
Loaded high intensity training, High intensity circuit training (HICT)
Other
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
11 Years to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Birmingham, Alabama
Completed No phase listed Observational Accepts healthy volunteers

UTSW HP [13-C] Pyruvate Injection in HCM

NCT03057002
Conditions
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis, Becker Muscular Dystrophy, Heart Failure With Preserved Ejection Fraction, Heart Failure With Reduced Ejection Fraction
Interventions
Hyperpolarized 13C-Pyruvate
Diagnostic Test
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Conditions
Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
Interventions
Screening
Other
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
448 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2015
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,733 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
41
States / cities
Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more
Conditions
Plasmacytoma, POEMS Syndrome
Interventions
Dexamethasone, Ixazomib Citrate, Lenalidomide, Questionnaire Administration
Drug · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2024
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Conditions
Muscular Dystrophy, Duchenne
Interventions
Eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
3
States / cities
Little Rock, Arkansas • Kansas City, Kansas • Richmond, Virginia
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
RAD001
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Duchenne Muscular Dystrophy
Interventions
RGX-202
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
1 Year and older · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
19
States / cities
Little Rock, Arkansas • Orange, California • Palo Alto, California + 16 more
Conditions
Familial Erythromelalgia
Interventions
BHV-7000, Placebo
Drug
Lead sponsor
Biohaven Therapeutics Ltd.
Industry
Eligibility
18 Years to 75 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
Interventions
Ataluren, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
21
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 17 more
Conditions
Genetic Disease, Communication
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
13 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Cerebral Palsy (CP), Stroke, Spinal Cord Injury, Traumatic Brain Injury, Charcot Marie Tooth Disease (CMT), Spina Bifida
Interventions
Participate in high intensity exercise of at least 20 minutes in 60 minutes session of RPE greater than 6.
Behavioral
Lead sponsor
University of Hartford
Other
Eligibility
8 Years to 99 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
West Hartford, Connecticut
Conditions
Neurofibromatosis 1, Chronic Pain
Interventions
iCanCope, iCanCope+Contingency Management
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 72 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Solid Tumors, Lung Cancer
Interventions
BKM120, Everolimus
Drug
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years and older
U.S. locations
1
States / cities
Portland, Oregon
Conditions
Muscular Dystrophy, Facioscapulohumeral
Interventions
Not listed
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
5 Years to 17 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
6
States / cities
Palo Alto, California • Iowa City, Iowa • Kansas City, Kansas + 3 more
Conditions
Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
Interventions
Pre-visit prep, usual care + exome seq
Behavioral · Diagnostic Test
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
0 Years and older
Enrollment
548 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
3
States / cities
Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts