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Recruiting Phase 1

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

NCT04626674 · tracked via the Priya Life Science USA tracker
Phase
Phase 1
Started
2020-11-23
Last updated
2026-09-11

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

delandistrogene moxeparvovec

delandistrogene moxeparvovec: Single IV infusion of delandistrogene moxeparvovec

Study 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.

Eligibility

Sex
MALE
Min age
2 Years
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

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

Trial sites (12)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas Recruiting
University of California, San Diego La Jolla California Recruiting
University of California, Los Angeles Los Angeles California Recruiting
Stanford University Palo Alto California Recruiting
University of California, Davis Sacramento California Recruiting
University of Florida Gainesville Florida Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
Washington University in St. Louis St Louis Missouri Recruiting
Duke University Medical Center Durham North Carolina Recruiting
Nationwide Children's Hospital Columbus Ohio Active Not Recruiting
Neurology Rare Disease Center Flower Mound Texas Recruiting
Children's Hospital of The King's Daughters Norfolk Virginia Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04626674 on ClinicalTrials.gov ↗ ← All trials in the USA