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Gene Therapy DMD Clinical Trials

16 studies · 13 recruiting in the DMD Warrior catalog.

These 16 trials are categorized under Gene Therapy in the DMD Warrior catalog — one of the main therapeutic approaches represented in contemporary Duchenne research.

13 studies are currently recruiting. Open any trial card for inclusion criteria, locations and registry links, or use the main trial search to combine this approach with age, country and phase filters.

Showing 16 clinical trials

Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys

Actively RecruitingPhase 3Gene Therapy

Age

6–10 years

Sponsor

Genethon

Countries

France +2 more

NCT ID

2023-505187-11-00

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Gene Delivery Study to Evaluate the Safety and Expression of Elevidys in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Active, Not RecruitingPhase 2Gene Therapy

Age

2–3 years

Sponsor

Hoffmann-La Roche

Countries

Belgium +5 more

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

Actively RecruitingObservationalGene Therapy

Age

0–25 years

Sponsor

REGENXBIO

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Solid Biosciences SGT-001 Gene Therapy Phase 1/2 Clinical Trial (IGNITE DMD)

Active, Not RecruitingPhase 1/2Gene Therapy

Age

4–17 years

Sponsor

Solid Biosciences

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

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

Actively RecruitingPhase 3Gene Therapy

Age

7–11 years

Sponsor

Solid Biosciences

Countries

United States +2 more

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Actively RecruitingPhase 1/2Gene Therapy

Age

0–17 years

Sponsor

Solid Biosciences

Countries

United States +3 more

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Evaluation of the Safety and Efficacy of BBM-D101 Gene Therapy to Treat Patients with Duchenne Muscular Dystrophy

Actively RecruitingEarly Phase 1Gene Therapy

Age

4–8 years

Sponsor

Shanghai Jiao Tong University School of Medicine

Countries

China

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) (ASCEND)

Actively RecruitingPhase 1Gene Therapy

Age

2–4 years

Sponsor

Insmed Gene Therapy LLC

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients

Actively RecruitingEarly Phase 1Gene Therapy

Age

2–7 years

Sponsor

Shanghai Siponuoyin Biotechnology Co Ltd

Countries

China

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)

Actively RecruitingPhase 2/3Gene Therapy

Age

1 years and older

Sponsor

REGENXBIO

Countries

United States, Canada

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy

Actively RecruitingPhase 2Gene Therapy

Age

4–9 years

Sponsor

GENERIUM

Countries

Russia, Belarus

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)

Not Yet RecruitingPhase 4Gene Therapy

Age

4 years and older

Sponsor

Sarepta Therapeutics

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Long-term Follow-up Study of Participants Who Received Elevidys in a Previous Clinical Study (EXPEDITION)

Enrolling by InvitationInterventionalGene Therapy

Age

2 years and older

Sponsor

Sarepta Therapeutics

Countries

United States +8 more

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Elevidys in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort (ENDEAVOR)

Actively RecruitingPhase 1Gene Therapy

Age

2 years and older

Sponsor

Sarepta Therapeutics

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)

Enrolling by InvitationObservationalGene Therapy

Age

4 years and older

Sponsor

Sarepta Therapeutics

Countries

United States

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Elevidys in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)

Active, Not RecruitingPhase 3Gene Therapy

Age

8 years and older

Sponsor

Sarepta Therapeutics

Countries

United States +13 more

NCT ID

This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.