drugset / Trial / NCT02310906
Phase I/II Study of SRP-4053 in DMD Patients
Phase 1/2
Completed
39 enrolled
Sarepta Therapeutics, Inc.
Catholic University of the Sacred Heart · collabConsultants for Research in Imaging and Spectroscopy · collabGreat Ormond Street Hospital for Children NHS Foundation Trust · collabInstitut de Myologie, France · collabRoyal Holloway University · collabSYSNAV · collabUniversity College, London · collabUniversity of Newcastle Upon-Tyne · collab
RandomizedParallel-groupQuadruple-blindTreatment
Summary
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
Timeline
- Start
- 2015-01-13
- Primary completion
- 2019-03-25
- Completion
- 2019-03-25
Outcome
Mixed primary results
paper Part 2 primary endpoints were dystrophin protein expression and 6-minute walk test (6MWT); PMID 34788571 ↗
paper Golodirsen increased dystrophin protein (16.0-fold; P < 0.001) PMID 34788571 ↗
paper At 3 years, 6MWT change from baseline was -99.0 m for golodirsen-treated patients versus -181.4 m for external controls (P = 0.067) PMID 34788571 ↗
Publications
- Background Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology. 2020 May 26;94(21):e2270-e2282. doi: 10.1212/WNL.0000000000009233. Epub 2020 Mar 5.
- Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Golodirsen | Unknown | 30 mg/kg | Intravenous |