NCT06597656

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years – 8 Years
• Healthy volunteers: Not accepted

Summary

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	delandistrogene moxeparvovec	Single IV infusion of delandistrogene moxeparvovec
PROCEDURE	Plasmapheresis	Therapeutic plasma exchange procedure

Timeline

• Start date: 2024-09-18
• Primary completion: 2025-08-05
• Completion: 2025-08-05
• First posted: 2024-09-19
• Last updated: 2025-09-04

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study