- Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy — Recruiting • Phase I / Phase II • NCT06138743.
- Gene therapy SRP-1003 tested for safety and effectiveness in patients with type 1 myotonic dystrophy.
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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. Conditions: Myotonic Dystrophy 1 Interventions: SRP-1003 IV Infusion, Placebo IV Infusion, SRP-1003 SC Injection, Placebo SC Injection Lead Sponsor: Sarepta…