Evaluating the Safety and Effects of SRP-1001 Compared to Placebo in Adolescents and Adults With Facioscapulohumeral Muscular Dystrophy Type 1

What this Trial Is About

Researchers are studying the safety, tolerability, pharmacokinetics, and pharmacodynamics of SRP-1001 in adolescents and adults with facioscapulohumeral muscular dystrophy Type 1 FSHD1. This study aims to evaluate the effects of SRP-1001 compared to placebo in two parts initially a single dose Part 1 followed by four doses Part 2. Participants who finish Part 1 may re-screen and be eligible for Part 2. The research includes detailed muscle biopsies guided by MRI before and after dosing. Participants receive either SRP-1001 or a placebo through intravenous infusion. In Part 1, participants get one dose, while in Part 2, they receive four doses. Muscle biopsies are collected before and after dosing in both parts, with an additional screening biopsy required for those entering Part 2. After completing Part 1 or Part 2, participants may join an open-label extension study or other future clinical trials. Throughout the study, participants undergo MRI-guided muscle biopsies, blood tests to assess drug levels and body responses, and safety monitoring for adverse events up to 90 days in Part 1 and up to 360 days in Part 2. Researchers track various pharmacokinetic measures like drug concentration and clearance. The study duration varies depending on the part, with ongoing monitoring to evaluate treatment effects and safety.

Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1