NCT03375164

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

Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 3 Months – 7 Years
• Healthy volunteers: Not accepted

Summary

This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	delandistrogene moxeparvovec	Single IV infusion of delandistrogene moxeparvovec.

Timeline

• Start date: 2018-01-04
• Primary completion: 2023-04-25
• Completion: 2023-04-25
• First posted: 2017-12-15
• Last updated: 2024-11-14
• Results posted: 2024-05-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study