A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

Plain-language summary

This study evaluates the safety of the gene transfer therapy delandistrogene moxeparvovec (SRP-9001) and how it produces dystrophin in the body, in patients with Duchenne muscular dystrophy (DMD). It enrolls patients with a definitive DMD diagnosis who fit various protocol-defined cohorts (ambulatory and non-ambulatory). The study is in Phase 1 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Recruiting

Sponsor

Sarepta Therapeutics, Inc.

Intervention

delandistrogene moxeparvovec

Start date

11/23/2020

Estimated completion

2/29/2028

Countries

United States

Related genes

Locations

• Little Rock, United States
• Palo Alto, United States
• Sacramento, United States
• St Louis, United States
• Columbus, United States
• Flower Mound, United States
• Norfolk, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.