Muscular Dystrophies: Clinical Trials

391 completed, 122 recruiting, 88 unknown, 71 terminated. 191 Phase 2, 180 Not applicable, 135 Phase 1, 83 Phase 3, 11 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	191	Completed 90, Terminated 35, Active not recruiting 23, Recruiting 18, Unknown 11, Enrolling by invitation 7, Withdrawn 4, Not yet recruiting 3
Not applicable	180	Completed 99, Unknown 26, Recruiting 25, Active not recruiting 8, Not yet recruiting 8, Enrolling by invitation 6, Withdrawn 5, Terminated 3
Phase 1	135	Completed 74, Recruiting 14, Terminated 13, Unknown 13, Active not recruiting 12, Withdrawn 5, Enrolling by invitation 2, Not yet recruiting 2
Phase 3	83	Completed 37, Terminated 18, Recruiting 10, Active not recruiting 6, Unknown 5, Withdrawn 4, Enrolling by invitation 3
Phase 4	11	Completed 4, Active not recruiting 2, Recruiting 2, Unknown 2, Terminated 1
Early Phase 1	9	Active not recruiting 3, Recruiting 3, Completed 2, Terminated 1

Follow Muscular Dystrophies: new trials by email Literature Review of Muscular Dystrophies trials Research Report on Muscular Dystrophies

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT) INTENT Neural Interface System - Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS)	Not applicable	Recruiting	Johns Hopkins University	5	2027-01-01	2026-10-09
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) forazapadin, Placebo Comparator - Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 2	Not yet recruiting	Satellos Bioscience, Inc.	51	2026-12-15	2026-10-13
Calcium Channel Blocker in Myotonic Dystrophy Type 1 Amlodipine - Myotonic Dystrophy 1	Phase 1	Not yet recruiting	University of Rochester	20	2026-12-07	2025-12-09
Urotherapy Education for Families of Children With Duchenne Muscular Dystrophy Urotherapy Education - Duchenne Muscular Dystrophy (DMD), Lower Urinary Tract Symptoms (LUTS)	Not applicable	Not yet recruiting	Lokman Hekim University	50	2026-11-01	2026-09-15
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting ELEVIDYS, Sirolimus, Glucocorticoids - Duchenne Muscular Dystrophy	Phase 4	Recruiting	Sarepta Therapeutics, Inc.	20	2026-10-30	2026-10-06
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension DM1, Myotonic Dystrophy		Recruiting	Virginia Commonwealth University	1000	2026-10-15	2026-10-16
iBCI Optimization for Veterans With Paralysis Mobile neural decoding platform (mobile iBCI) - Spinal Cord Injury, Amyotrophic Lateral Sclerosis	Not applicable	Not yet recruiting	VA Office of Research and Development	2	2026-10-02	2026-07-27
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy KER-065 - Duchenne Muscular Dystrophy	Phase 2	Recruiting	Keros Therapeutics, Inc.	36	2026-09-25	2026-09-30
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function Becker Muscular Dystrophy		Not yet recruiting	Institut de Myologie, France	75	2026-09-15	2026-07-31
Intermittent vs Alternate Daily Regimen of Prednisolone in Ambulatory Boys With Duchenne Muscular Dystrophy Alternate-day prednisolone, Intermittent prednisolone - Duchenne Muscular Dystrophy	Not applicable	Not yet recruiting	Muhammad Aamir Latif	40	2026-09-15	2026-08-26
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy Semaglutide (Rybelsus®), Placebo - Duchenne Muscular Dystrophy (DMD)	Phase 1 / Phase 2	Not yet recruiting	Vanderbilt University Medical Center	30	2026-09-01	2026-06-11
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)	Phase 3	Recruiting	Avidity Biosciences, Inc.	70	2026-09-01	2026-10-13
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD SFL-0821 for injection, Placebo - Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 1 / Phase 2	Recruiting	Soufflé Therapeutics, Inc.	90	2026-09-01	2026-10-30
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy Multicomponent Intervention - Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA)	Not applicable	Active not recruiting	Aga Khan University	34	2026-08-15	2026-10-09
An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period Myotonic Dystrophy 1, Myotonic Dystrophy 2		Not yet recruiting	Lupin Ltd.	100	2026-08-03	2026-07-28
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE) ENTR-601-44, ENTR-601-45 - Duchenne Muscular Dystrophy (DMD)	Phase 2	Not yet recruiting	Entrada Therapeutics, Inc.	80	2026-08-01	2026-07-02
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength - Duchenne Muscular Dystrophy (DMD)	Not applicable	Not yet recruiting	Centre Hospitalier Universitaire de Liege	50	2026-07-30	2026-06-23
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD Apitegromab, Placebo - Facioscapulohumeral Muscular Dystrophy, FSHD	Phase 2	Recruiting	Scholar Rock, Inc.	60	2026-07-30	2026-09-02
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients VM100 Nutritional Supplement - Duchenne Muscular Dystrophy, Duchenne Disease	Phase 2	Recruiting	University of Florida	20	2026-07-27	2026-10-08
Registry for Duchenne and Becker Muscular Dystrophy Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy		Recruiting	Dr. Andreas Ziegler	1500	2026-07-13	2026-10-08

Showing 20 of 811. Sign in to see all of them.

Narrower conditions

Muscular Dystrophy, Duchenne (479)  ·  Myotonic Dystrophy (124)  ·  Muscular Dystrophy, Facioscapulohumeral (75)  ·  Neuromuscular Diseases (72)  ·  Muscular Dystrophies, Limb-Girdle (46)  ·  Muscular Diseases (40)  ·  Muscular Atrophy, Spinal (30)  ·  Amyotrophic Lateral Sclerosis (26)  ·  Musculoskeletal Diseases (26)  ·  Spinal Cord Injuries (25)  ·  Genetic Diseases, Inborn (22)  ·  Nervous System Diseases (21)  ·  Motor Activity (20)  ·  Cardiomyopathies (17)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (810)  ·  Muscular Disorders, Atrophic (795)  ·  Nervous System Diseases (790)  ·  Genetic Diseases, Inborn (789)  ·  Musculoskeletal Diseases (785)  ·  Muscular Diseases (771)  ·  Neuromuscular Diseases (739)  ·  Genetic Diseases, X-Linked (479)  ·  Neurodegenerative Diseases (179)  ·  Heredodegenerative Disorders, Nervous System (144)  ·  Myotonic Disorders (123)  ·  Central Nervous System Diseases (84)  ·  Pathological Conditions, Signs and Symptoms (82)  ·  Nutritional and Metabolic Diseases (65)

Interventions

ataluren (13)  ·  eteplirsen (12)  ·  Prednisone (11)  ·  Exercise (10)  ·  VBP15 compound (9)  ·  deflazacort (8)  ·  Tadalafil (7)  ·  Sodium Chloride (6)  ·  givinostat (6)  ·  givinostat hydrochloride (6)  ·  idebenone (6)  ·  Adrenal Cortex Hormones (5)  ·  casimersen (5)  ·  golodirsen (5)  ·  viltolarsen (5)

Sponsors

Sarepta Therapeutics, Inc. (38)  ·  Hacettepe University (22)  ·  PTC Therapeutics (19)  ·  Cooperative International Neuromuscular Research Group (16)  ·  Assistance Publique - Hôpitaux de Paris (14)  ·  Copenhagen University Hospital, Rigshospitalet (14)  ·  Avidity Biosciences, Inc. (11)  ·  BioMarin Pharmaceutical (11)  ·  University of Rochester (11)  ·  Nationwide Children's Hospital (10)  ·  University of Florida (10)  ·  Virginia Commonwealth University (10)  ·  NS Pharma, Inc. (9)  ·  Santhera Pharmaceuticals (9)  ·  Centre Hospitalier Universitaire de Nice (8)

Countries

United States (372)  ·  France (128)  ·  Italy (121)  ·  United Kingdom (121)  ·  Canada (113)  ·  Spain (80)  ·  Turkey (Türkiye) (78)  ·  Germany (69)  ·  Belgium (67)  ·  Netherlands (61)  ·  Australia (56)  ·  Denmark (41)  ·  Israel (35)  ·  Japan (31)  ·  Sweden (31)

Top investigators

Nicholas Johnson, MD (6)  ·  Craig McDonald, MD (5)  ·  Kevin Flanigan, MD (5)  ·  Paula R Clemens, MD (5)  ·  Ronald G Victor, MD (5)  ·  Diana M Escolar, MD (4)  ·  Dirk Fischer, MD (4)  ·  Francesco Bibbiani, MD (4)  ·  Mark Awadalla (4)  ·  Carlos BM Monteiro, Ph.D. (3)

Results publications

PMID 32956407, PMID 37539981, PMID 41671094, PMID 42613652, PMID 26573217, PMID 32434278, PMID 32522498, PMID 32851872, PMID 34693725, PMID 34779542, PMID 35794169, PMID 38190001, PMID 38521535, PMID 38753764, PMID 40152935, PMID 41058127, PMID 41774261, PMID 42139656, PMID 42202243, PMID 10826123, PMID 10836341, PMID 11751186, PMID 11972163, PMID 12030525, PMID 12122118, PMID 12210323, PMID 12351998, PMID 12409823, PMID 1281472, PMID 13997180, PMID 14510658, PMID 14760317, PMID 15293280, PMID 15302625, PMID 15781190, PMID 1580445, PMID 15984021, PMID 16055882, PMID 16267188, PMID 16602102, PMID 16763895, PMID 16763896, PMID 16849828, PMID 16900450, PMID 17029646, PMID 17136445, PMID 17336067, PMID 17385269, PMID 17389552, PMID 17450125

NIH funding

2023 (12), 2015 (8), 2019 (7), 2025 (6), 2016 (5), 2017 (4), 2014 (3), U54NS048843 (3), R44NS095423 (2), U34AR068616 (2), 1DP2DC021055 (1), 2008 (1), 2009 (1), 2018 (1), I01RX003803 (1)