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delandistrogene moxeparvovec

GENETIC10 trials

Sponsors

Sarepta Therapeutics, Inc., Hoffmann-La Roche

Conditions

Duchenne Muscular DystrophyMuscular Dystrophy, Duchenne

Phase 1

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
CompletedNCT03375164
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2018-01-04End: 2023-04-25Updated: 2024-11-14
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
CompletedNCT03769116
Sarepta Therapeutics, Inc.Muscular Dystrophy, Duchenne
Start: 2018-12-05End: 2023-08-16Updated: 2024-11-14
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
RecruitingNCT04626674
Sarepta Therapeutics, Inc.Muscular Dystrophy, Duchenne
Start: 2020-11-23End: 2028-02-29Target: 83Updated: 2026-03-04
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
TerminatedNCT06241950
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2024-01-29End: 2025-10-10Updated: 2025-11-20
A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74
TerminatedNCT06597656
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2024-09-18End: 2025-08-05Updated: 2025-09-04

Phase 2

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
Active, not recruitingNCT06128564
Hoffmann-La RocheDuchenne Muscular Dystrophy
Start: 2023-11-29End: 2034-01-30Updated: 2026-02-10

Phase 3

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
CompletedNCT05096221
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2021-10-27End: 2024-10-25Updated: 2025-07-08
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Active, not recruitingNCT05881408
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2023-05-31End: 2028-06-30Target: 148Updated: 2025-06-22
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Enrolling by invitationNCT05967351
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2023-09-27End: 2033-10-31Target: 400Updated: 2026-02-06

Unknown Phase

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
Enrolling by invitationNCT06270719
Sarepta Therapeutics, Inc.Duchenne Muscular Dystrophy
Start: 2024-02-07End: 2038-12-31Target: 500Updated: 2025-12-02

Related Papers

Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.
2026-01-10
Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial.
2025-11-03
Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy
JAMA Neurology2025-05-123 citations
Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy
Scientific Reports2025-01-0216 citations
Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy
Neurology and Therapy2024-11-264 citations
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
Nature Medicine2024-10-09103 citations
D.4 Safety and efficacy of delandistrogene moxeparvovec versus placebo in Duchenne muscular dystrophy (EMBARK): Pivotal Phase 3 primary results
Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques2024-05-23
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
PLoS ONE2024-05-162 citations
Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy.
2024-01-0518 citations
Long‐term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial
Muscle & Nerve2023-08-1465 citations
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to &lt;8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study <scp>SRP</scp>‐9001‐103 (<scp>ENDEAVOR</scp>)
Annals of Neurology2023-08-0482 citations
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy
Frontiers in Cell and Developmental Biology2023-07-1168 citations
EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP- 9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics (P5-8.012)
Neurology2023-04-257 citations
A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) (S48.004)
Neurology2023-04-257 citations
One-year Data from ENDEAVOR, a Phase 1b Trial of Delandistrogene Moxeparvovec (SRP-9001) in Patients with Duchenne Muscular Dystrophy (DMD) (S48.003)
Neurology2023-04-252 citations
Long-term Safety and Efficacy in Patients with DMD 4 Years Post-Treatment with Delandistrogene Moxeparvovec (SRP-9001) in a Phase 1/2a Study (P3-8.006)
Neurology2023-04-252 citations
Analysis of Vector Shedding Following Treatment with Delandistrogene Moxeparvovec (SRP-9001), an Investigational rAAVrh74-Based Gene Therapy for Duchenne Muscular Dystrophy (DMD) (P5-8.013)
Neurology2023-04-251 citations
Integrated Analyses of Data from Clinical Trials of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD) (S48.006)
Neurology2023-04-25
Phase 1/2a Trial of SRP-9001 in Patients with Duchenne Muscular Dystrophy: 3-Year Safety and Functional Outcomes (S23.004)
Neurology2022-05-031 citations
A Phase 2 Clinical Trial Evaluating the Safety and Efficacy of SRP-9001 for Treating Patients with Duchenne Muscular Dystrophy (S23.002)
Neurology2022-05-03

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