Clinical Trial

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

Recruiting Phase 1/2
View on ClinicalTrials.gov →
Summary
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Protocol Amendment History 5 changes
critical Primary endpoint(s) modified 2026-08-12
notable Sponsor changed: Arrowhead Pharmaceuticals -> Sarepta Therapeutics, Inc. 2026-08-12
notable Trial sites expanded: 11 -> 35 locations 2026-08-12
notable Primary completion pushed: 2026-12 -> 2026-12-31 2026-08-12
minor Completion pushed: 2026-12 -> 2026-12-31 2026-08-12
Trial Details
NCT Number NCT06138743
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Myotonic Dystrophy 1
Enrollment 78 participants
Start Date 2024-03-04
Primary Completion 2026-12-31 (estimated)
Study Completion 2026-12-31 (estimated)
Updated on ClinicalTrials.gov 2026-08-11