Recruiting
Phase 1

Delandistrogene Moxeparvovec

Sponsor:

Sarepta Therapeutics, Inc.

Code:

NCT04626674

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Interventions

delandistrogene moxeparvovec

Study Details

Brief summary:

Cohort 8 (non-ambulatory participants) is currently enrolling new participants. Enrollment for Cohorts 1 through 7 has been completed.

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with Duchenne Muscular Dystrophy (DMD). The maximum participant duration for this study is 156 weeks.

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT04626674

Start date

Nov 23, 2020

Status verified date

Jun, 2026

Completion date

Feb 29, 2028

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
  • Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥3 at the Screening visit and has a total PUL score of ≥20 and ≤40 at the time of Screening.
  • Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
  • Cohort 1: Is ambulatory, and ≥4 to <8 years of age at the time of Screening.
  • Cohort 2: Is ambulatory, and ≥8 to <18 years of age at the time of Screening.
  • Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.
  • Cohort 4: Is ambulatory and ≥3 to <4 years of age at the time of Screening.
  • Cohort 5a: Is ambulatory and ≥4 to <9 years of age with time to rise from the floor ≤7 seconds at the screening visit.
  • Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.
  • Cohort 6: Is ambulatory, and ≥2 to <3 years of age at the time of Screening.
  • Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.
  • Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
  • Genetic mutation inclusion criteria vary by cohort.

All Cohorts:

  • Ability to cooperate with motor assessment testing.
  • rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.

Exclusion Criteria:

  • Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
  • Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer.
  • Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits.
  • Abnormality in protocol-specified diagnostic evaluations or laboratory tests.

Note: Other inclusion/exclusion criteria apply.

Study Design

Enrollment

83 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Delandistrogene Moxeparvovec

Participants will receive a single intravenous (IV) infusion of delandistrogene moxeparvovec on Day 1.

Interventions

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec

Primary outcome measure

  • Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot [ Time Frame: Baseline, Week 12 ]
  • Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot [ Time Frame: Week 12 ]
  • Cohort 8: Number of Participants with Acute Liver Injury (ALI) [ Time Frame: Baseline up to Week 72 ]

Central Contacts and Locations

Central contacts

Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4

1-888-SAREPTA (1-888-727-3782)SareptAlly@Sarepta.com

Locations

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Contacts

501-364-1868KurakuVV@archildrens.org

Principal Investigator:

Aravindhan Veerapandiyan, MD

Stanford University

Recruiting

Palo Alto, California, United States, 94304

Contacts

neuromuscularresearch@stanford.edu

Principal Investigator:

John Day, MD

University of California, Davis

Recruiting

Sacramento, California, United States, 95616

Principal Investigator:

Craig McDonald, MD

Washington University in St. Louis

Recruiting

St Louis, Missouri, United States, 63110

Contacts

314-362-6981NeuromusclePediatricResearch@wustl.edu

Principal Investigator:

Craig Zaidman, MD

Neurology Rare Disease Center

Recruiting

Flower Mound, Texas, United States, 75028

Contacts

972-999-1011Research@neuromdcenter.com

Principal Investigator:

Diana Castro, MD

Children's Hospital of The King's Daughters

Recruiting

Norfolk, Virginia, United States, 23507

Contacts

Proud.Research@chkd.org

Principal Investigator:

Crystal Proud, MD

More Information

Sponsor

Sarepta Therapeutics, Inc.

Last update posted

Jun 24, 2026

Last verified

Jun, 2026

Keywords

  • Duchenne Muscular Dystrophy
  • Gene-Delivery
  • Gene Therapy
  • DMD
  • Ambulatory Non-ambulatory
  • Pediatric
  • Adult
  • Dystrophin

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Sarepta Therapeutics, Inc. on 2026-06-24.