Delandistrogene moxeparvovec gene therapy for Duchenne muscular dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an open-label Phase 1 gene transfer trial focused on the safety of and expression from delandistrogene moxeparvovec in participants with genetically confirmed Duchenne muscular dystrophy. The study enrolls multiple cohorts spanning ambulatory and non-ambulatory participants across pediatric and adolescent age ranges, with cohort-specific entry criteria (including an upper-limb performance requirement for Cohort 8). Participants receive the gene-delivery intervention and are followed for up to 156 weeks for safety, laboratory, and expression endpoints. Enrollment for Cohorts 1–7 is complete and Cohort 8 is currently enrolling at several U.S. academic medical centers.

Why it matters

Who can join: the full rules

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 ≥2 at the Screening visit and has a total PUL score of ≥12 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.

Where it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• University of California, San Diego
  La Jolla, California, United States Recruiting
• University of California, Los Angeles
  Los Angeles, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• University of California, Davis
  Sacramento, California, United States Recruiting
• University of Florida
  Gainesville, Florida, United States Recruiting

Study contacts

• Study coordinator: Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
• Email: SareptAlly@Sarepta.com
• Phone: 1-888-SAREPTA (1-888-727-3782)

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.