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Dystrophy Clinical Trials

Browse 493 Dystrophy clinical trials enrolling across the United States and Canada. Search by city or state to find one near you.

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Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD)

for
Neovascular Age-related Macular Degeneration
Location: Portland ME, Little Silver NJ, Philadelphia PA,
Sponsor: AbbVie

Sex: All

Age: 50+

Code: NCT07007065

Phase 3, Recruiting
Featured

A Study Assessing Corneal Endothelial Cells in Participants With Neovascular Age-related Macular Degeneration (nAMD) Treated With the Port Delivery System With Ranibizumab (PDS)

for
Neovascular Age-related Macular Degeneration
Location: Hickory NC, Austin TX
Sponsor: Genentech, Inc.

Sex: All

Age: 50+

Code: NCT04853251

Recruiting
Active & Responsive

Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)

for
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne,
Location: Hillsborough NC
Sponsor: Dyne Therapeutics

Sex: Male

Age: 4 - 18

Code: NCT07608432

Phase 3, Recruiting
Active & Responsive

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

for
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS),
Location: Baltimore MD
Sponsor: Johns Hopkins University

Sex: All

Age: 18 - 70+

Code: NCT07521930

Recruiting
Active & Responsive

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD

for
Duchenne Muscular Dystrophy
Location: Nashville TN
Sponsor: Vanderbilt University Medical Center

Sex: Male

Age: 10+

Code: NCT06124196

Recruiting

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Active & Responsive

Trial of Cell Based Therapy for DMD

for
Duchenne Muscular Dystrophy
Location: Minneapolis MN
Sponsor: Masonic Cancer Center, University of Minnesota

Sex: All

Age: 18+

Code: NCT06692426

Phase 1, Recruiting
Active & Responsive

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy

for
Duchenne Muscular Dystrophy
Location: Ottawa, Toronto, London, Vancouver
Sponsor: Italfarmaco

Sex: Male

Age: 9 - 17

Code: NCT05933057

Phase 3, Recruiting
Active & Responsive

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

for
Spinal Muscular Atrophy Type 3,
Location: Boston MA, New York NY, Palo Alto CA
Sponsor: Columbia University

Sex: All

Age: 5+

Code: NCT06839469

Recruiting
Active & Responsive

Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)

for
Stargardt Disease, Stargardt Disease 1, Cone Rod Dystrophy,
Location: Boston MA, Gainesville FL, Ann Arbor MI, Cincinnati OH,
Sponsor: Ascidian Therapeutics, Inc

Sex: All

Age: 5+

Code: NCT06445322

Recruiting
Active & Responsive

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

for
Duchenne Muscular Dystrophy
Location: Toronto, Calgary, Vancouver
Sponsor: Solid Biosciences Inc.

Sex: Male

Age: 7 - 11

Code: NCT07160634

Phase 3, Recruiting
Active & Responsive

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

for
Facioscapulohumeral Muscular Dystrophy, FSHD
Location: Austin TX
Sponsor: Scholar Rock, Inc.

Sex: All

Age: 18 - 60

Code: NCT07435129

Phase 2, Recruiting
Active & Responsive

Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy

for
Duchenne Muscular Dystrophy (DMD)
Location: Nashville TN
Sponsor: Vanderbilt University Medical Center

Sex: Male

Age: 8 - 18

Code: NCT07172971

Phase 1, Recruiting
Active & Responsive

Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)

for
Muscular Dystrophy, Facioscapulohumeral
Location: Rochester NY, Durham NC, Kansas City KS
Sponsor: University of Kansas Medical Center

Sex: All

Age: 5 - 17

Code: NCT06847282

Recruiting
Active & Responsive

An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)

for
Myotonic Dystrophy 1
Location: Saguenay, Ottawa, Calgary
Sponsor: PepGen Inc

Sex: All

Age: 0+

Code: NCT07220603

Phase 2, Recruiting
Active & Responsive

Urinary Titin Biomarker in DMD

for
Duchenne Muscular Dystrophy (DMD),
Location: Philadelphia PA
Sponsor: Children's Hospital of Philadelphia

Sex: Male

Age: 2 - 10

Code: NCT07332013

Recruiting
Active & Responsive

Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients

for
Duchenne Muscular Dystrophy, Duchenne Disease,
Location: Gainesville FL
Sponsor: University of Florida

Sex: Male

Age: 6+

Code: NCT07766980

Phase 2, Recruiting
Active & Responsive

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

for
Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy,
Location: Baltimore MD, Richmond VA, Rochester NY, Gainesville FL,
Sponsor: Dyne Therapeutics

Sex: All

Age: 16+

Code: NCT07486934

Phase 3, Recruiting
Active & Responsive

Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

for
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\),
Location: Boston MA, Bethesda MD, Rochester NY, Pittsburgh PA,
Sponsor: Nationwide Children's Hospital

Sex: All

Age: 0 - 5

Code: NCT06503367

Recruiting
Active & Responsive

Clenbuterol to Target DUX4 in FSHD

for
Muscular Dystrophy, Facioscapulohumeral
Location: Rochester NY, Kansas City KS, Seattle WA
Sponsor: Jeffrey Statland

Sex: All

Age: 18 - 70+

Code: NCT06721299

Phase 1, Recruiting
Active & Responsive

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

for
Congenital Myotonic Dystrophy, CDM
Location: Richmond VA, Rochester NY, Fairway KS, Los Angeles CA
Sponsor: Virginia Commonwealth University

Sex: All

Age: 0 - 4

Code: NCT05224778

Recruiting