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

Summary

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Timeline

Start

2021-10-27

Primary completion

2023-10-04

Completion

2024-10-25

Publications

• Results Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z. Epub 2024 Oct 9.
• Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Furgerson M, Ding K, Singh P, Potter R, Asher DR, Murphy AP, Reid C, Hooper G, Torre CO, Manfrini M, Rodino-Klapac LR. Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial. Neurol Ther. 2026 Apr;15(2):545-559. doi: 10.1007/s40120-025-00879-8. Epub 2026 Jan 10.
• Audhya I, Nacson AB, Gooch K, Basnyat B, Slota C, Martin S, Murphy A, Lansdall CJ, Ciobanu T, Nascimento A, Veerapandiyan A. Caregiver-reported Patient Experiences with Duchenne Muscular Dystrophy: Qualitative In-trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial. Neurol Ther. 2026 Feb;15(1):41-60. doi: 10.1007/s40120-025-00842-7. Epub 2025 Nov 3.
• Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR. Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: An Exploratory Analysis From the EMBARK Randomized Clinical Trial. JAMA Neurol. 2025 Jul 1;82(7):734-744. doi: 10.1001/jamaneurol.2025.0992.
• McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I; EMBARK Study Group. Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. Neurol Ther. 2025 Feb;14(1):211-225. doi: 10.1007/s40120-024-00685-8. Epub 2024 Nov 26.

Drugs

Indications