Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 21, 2026, 6:34 PM EDT

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Showing 1–24 of 479 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Mitochondrial Disease, Chronic Kidney Disease
Interventions
Probucol
Drug
Lead sponsor
RiboNova Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Muscular Dystrophy, Duchenne
Interventions
Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle, Echocardiogram
Other
Lead sponsor
University of Florida
Other
Eligibility
5 Years to 15 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Completed Phase 3 Interventional Results available

Study of Eteplirsen in DMD Patients

NCT02255552
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
eteplirsen
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 16 Years · Male only
Enrollment
109 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
37
States / cities
Phoenix, Arizona • Los Angeles, California • Sacramento, California + 32 more
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Sacramento, California • Chicago, Illinois • Durham, North Carolina + 1 more
Conditions
Duchenne Muscular Dystrophy
Interventions
Tadalafil, Sildenafil
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
7 Years to 15 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Muscular Dystrophy, Duchenne
Interventions
Creatine Monohydrate, Glutamine
Drug
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
5 Years to 9 Years · Male only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone, Prednisone, Placebo
Drug · Other
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
121 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
10
States / cities
Davis, California • Los Angeles, California • Aurora, Colorado + 7 more
Conditions
Muscular Dystrophies
Interventions
rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
Genetic
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
5 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Myotonic Dystrophy 1, DM1
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Denver, Colorado + 7 more
Conditions
Duchenne Muscular Dystrophy
Interventions
No intervention
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Richmond, Virginia
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, SRP-4053, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
6 Years to 13 Years · Male only
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
23
States / cities
Phoenix, Arizona • Los Angeles, California • San Diego, California + 19 more
Conditions
Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Month to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Katherine Mathews
Other
Eligibility
Not listed
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2030
U.S. locations
1
States / cities
Iowa City, Iowa
Conditions
Duchenne Muscular Dystrophy
Interventions
Ezutromid
Drug
Lead sponsor
Summit Therapeutics
Industry
Eligibility
5 Years and older · Male only
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
9
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 6 more
Active, not recruiting No phase listed Observational

Defining Endpoints in Becker Muscular Dystrophy

NCT05257473
Conditions
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years and older · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Iowa City, Iowa + 6 more
Completed Early Phase 1 Interventional Accepts healthy volunteers

Nitric Oxide Production in MELAS Syndrome

NCT01339494
Conditions
MELAS Syndrome
Interventions
Arginine and citrulline supplementations
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
3 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
onasemnogene abeparvovec
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
0 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
1
States / cities
Norfolk, Virginia
Conditions
Muscular Dystrophy, Duchenne
Interventions
SRP-5051
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
7
States / cities
Sacramento, California • Gulf Breeze, Florida • Atlanta, Georgia + 4 more
Conditions
Duchenne Muscular Dystrophy
Interventions
ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Houston, Texas
Conditions
Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases, Neuromuscular Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn, Nervous System Diseases
Interventions
Deramiocel (CAP-1002), Placebo
Biological
Lead sponsor
Capricor Inc.
Industry
Eligibility
10 Years and older · Male only
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
20
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 17 more
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Conditions
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophies
Interventions
BrainGate Neural Interface System
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Stanford, California • Boston, Massachusetts