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)

NCT06241950 ↗

Phase 1 Terminated 5 enrolled Sarepta Therapeutics, Inc. Hansa Biopharma AB · collab

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2024-01-29
Primary completion: 2025-10-10
Completion: 2025-10-10

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Imlifidase	Protein / enzyme biologic	—	Intravenous
Subject	delandistrogene moxeparvovec	Gene therapy (AAV / viral vector)	—	Intravenous

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)

Summary

Timeline

Drugs

Indications