NCT06241950

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years – 9 Years
• Healthy volunteers: Not accepted

Summary

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	delandistrogene moxeparvovec	Single IV infusion of delandistrogene moxeparvovec
BIOLOGICAL	imlifidase	IV infusion of Imlifidase

Timeline

• Start date: 2024-01-29
• Primary completion: 2025-10-10
• Completion: 2025-10-10
• First posted: 2024-02-05
• Last updated: 2025-11-20

Locations

1 site across 1 country: Spain

Regulatory

• FDA-regulated drug study