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ClinicalTrials.gov public records Last synced May 22, 2026, 1:44 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Muscular Dystrophy Clear all
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy (FSHD)
Interventions
Losmapimod
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
Irvine, California • Los Angeles, California • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced May 22, 2026, 1:44 AM EDT
Withdrawn Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
Biological · Other
Lead sponsor
Kevin Flanigan
Other
Eligibility
9 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 5, 2018 · Synced May 22, 2026, 1:44 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 45 Years
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
10
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 7 more
Source: ClinicalTrials.gov public record
Updated May 27, 2025 · Synced May 22, 2026, 1:44 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
Interventions
Not listed
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Up to 5 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Los Angeles, California • Stanford, California • Chicago, Illinois + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 22, 2026, 1:44 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
Interventions
AOC 1001, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced May 22, 2026, 1:44 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,323 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced May 22, 2026, 1:44 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Interventions
ACE-083
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
19
States / cities
Orange, California • Sacramento, California • Aurora, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2022 · Synced May 22, 2026, 1:44 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Lymphoma, Large B-Cell, Diffuse
Interventions
lenalidomide, Placebo, Rituximab, Cyclophosphamide, Doxorubicin, prednisone, vincristine
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 80 Years
Enrollment
570 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
22
States / cities
Fayetteville, Arkansas • Orange, California • Sacramento, California + 16 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2023 · Synced May 22, 2026, 1:44 AM EDT
Terminated Phase 3 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
idebenone 150 mg film-coated tablets
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
11 Years and older · Male only
Enrollment
161 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
13
States / cities
Birmingham, Alabama • Tucson, Arizona • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2021 · Synced May 22, 2026, 1:44 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
327 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
5
States / cities
Aurora, Colorado • Boston, Massachusetts • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2016 · Synced May 22, 2026, 1:44 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Interventions
Pitolisant Oral Tablet, Placebo oral tablet
Drug
Lead sponsor
Harmony Biosciences Management, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Irvine, California • Aurora, Colorado • Tampa, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced May 22, 2026, 1:44 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 22, 2026, 1:44 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Tadalafil
Drug
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 13 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 26, 2025 · Synced May 22, 2026, 1:44 AM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced May 22, 2026, 1:44 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
Interventions
Not listed
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced May 22, 2026, 1:44 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Limb Girdle Muscular Dystrophy
Interventions
SRP-6004
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 22, 2026, 1:44 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
givinostat, placebo
Drug
Lead sponsor
Italfarmaco
Industry
Eligibility
6 Years to 17 Years · Male only
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
13
States / cities
Davis, California • San Diego, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2023 · Synced May 22, 2026, 1:44 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
MNK-1411, Placebo
Drug · Other
Lead sponsor
Mallinckrodt ARD LLC
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Gulf Breeze, Florida • Atlanta, Georgia • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced May 22, 2026, 1:44 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Motor Neuron Disease, Muscular Disease, Muscular Dystrophy, Peripheral Nervous System Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years to 120 Years
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced May 22, 2026, 1:44 AM EDT
Recruiting No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced May 22, 2026, 1:44 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
VX-670, Placebo
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 64 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
San Carlos, California • Gainesville, Florida • Fairway, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced May 22, 2026, 1:44 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Viltolarsen, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 10, 2024 · Synced May 22, 2026, 1:44 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 22, 2026, 1:44 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced May 22, 2026, 1:44 AM EDT