Background illustration for clinical trials search

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.

Filter
1

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

for
ARB, BVMD, Autosomal-Dominant Bestrophinopathy,
Location: New York NY, Cincinnati OH, Dallas TX, Los Angeles CA
Sponsor: Opus Genetics, Inc

Sex: All

Age: 18+

Code: NCT07185256

Phase 1, Phase 2, Recruiting

Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata

for
Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (Apeced),
Location: Bethesda MD
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Sex: All

Age: 12 - 70+

Code: NCT05398809

Phase 2, Recruiting

The Baby Duchenne Study: Characterizing Developmental and Clinical Outcomes in the First Three Years in Children With Duchenne Muscular Dystrophy

for
Duchenne Muscular Dystrophy (DMD)
Location: Rochester NY, Columbus OH
Sponsor: University of Rochester

Sex: Male

Age: 0 - 3

Code: NCT07092540

Recruiting

DT-168 in Keratoplasty Patients With Fuchs Endothelial Corneal Dystrophy

for
Fuchs Endothelial Corneal Dystrophy, Fuchs
Location: Grand Rapids MI, Indianapolis IN
Sponsor: Design Therapeutics, Inc.

Sex: All

Age: 30+

Code: NCT07024693

Phase 2, Recruiting

The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration

for
Alcohol Use Disorder, Retinal Dystrophies,
Location: Rochester NY
Sponsor: University of Rochester

Sex: All

Age: 18+

Code: NCT06319872

Phase 1, Recruiting

Interested in new trials?

Get alerts for new trials in your area by subscribing now

Subscriber Image

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

for
Myotonic Dystrophy
Location: New York NY, Montreal, Philadelphia PA, Richmond VA,
Sponsor: Sanofi

Sex: All

Age: 10 - 55

Code: NCT06844214

Phase 1, Phase 2, Recruiting

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

for
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Location: Gainesville FL, Portland OR
Sponsor: University of Florida

Sex: Male

Age: 5 - 62

Code: NCT01484678

Recruiting

EyeConic: Qualification for Cone-Optogenetics

for
Retinal Dystrophies
Location: Miami FL, Pittsburgh PA, San Francisco CA
Sponsor: Institute of Molecular and Clinical Ophthalmology Basel

Sex: All

Age: 0+

Code: NCT05294978

Recruiting

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

for
Myotonic Dystrophy Type 1 (DM1), DM1
Location: Rochester NY
Sponsor: University of Rochester

Sex: All

Age: 18 - 70+

Code: NCT07505342

Recruiting

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

for
Primary Photoreceptor Disease, Retinitis Pigmentosa (RP),
Location: Philadelphia PA, Miami FL, Madison WI, Dallas TX,
Sponsor: BlueRock Therapeutics

Sex: All

Age: 18+

Code: NCT06789445

Phase 1, Phase 2, Recruiting

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

for
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries,
Location: Sacramento CA
Sponsor: Leigh R. Hochberg, MD, PhD.

Sex: All

Age: 18 - 70+

Code: NCT06094205

Recruiting

Stargardt-like Macular Dystrophy (STDG3) Secondary to Mutations in ELOVL4

for
Stargardt-Like Macular Dystrophy
Location: Bethesda MD
Sponsor: National Eye Institute (NEI)

Sex: All

Age: 10 - 70+

Code: NCT04591483

Recruiting

Study of ATX-01 in Participants With DM1

for
Myotonic Dystrophy 1
Location: Chicoutimi, Richmond VA, Gainesville FL, Iowa City IA,
Sponsor: ARTHEx Biotech S.L.

Sex: All

Age: 18 - 64

Code: NCT06300307

Phase 1, Phase 2, Recruiting

National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

for
Inherited Ophthalmic Diseases, Hypopigmentation Disorder,
Location: Bethesda MD
Sponsor: National Eye Institute (NEI)

Sex: All

Age: 0 - 70+

Code: NCT06491615

Recruiting

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

for
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy,
Location: Richmond VA
Sponsor: Virginia Commonwealth University

Sex: All

Age: 3 - 17

Code: NCT06747884

Recruiting

Once Weekly Infant Corticosteroid Trial for DMD

for
Duchenne Muscular Dystrophy
Location: Columbus OH, Cincinnati OH, Chicago IL, Dallas TX
Sponsor: Anne M. Connolly

Sex: Male

Age: 0 - 2

Code: NCT05412394

Recruiting

Glanatec(R) for Descemet Stripping in Fuch's Endothelial Dystrophy

for
Fuchs' Endothelial Dystrophy
Location: Glenview IL
Sponsor: Marian Macsai, MD

Sex: All

Age: 18 - 70+

Code: NCT03249337

Recruiting

Comparing Endoglide to Endoserter for DSAEK Graft Insertion

for
Fuch's Endothelial Dystrophy,
Location: Toronto
Sponsor: University Health Network, Toronto

Sex: All

Age: 18+

Code: NCT01791075

Recruiting

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

for
Stargardt Disease, Stargardt Macular Degeneration,
Location: Boston MA, Miami FL, Dallas TX, Aurora CO, Portland OR
Sponsor: Splice Bio

Sex: All

Age: 12 - 65

Code: NCT06942572

Phase 1, Phase 2, Recruiting

DMD Voice: Qualitative Interviews With Patients and Caregivers

for
Duchenne Muscular Dystrophy (DMD)
Location: Yardley PA
Sponsor: Red Nucleus Enterprise Solutions, LLC

Sex: Male

Age: 10+

Code: NCT06925269

Recruiting