Phase 12a Study Evaluating Safety and Effects of SRP-1003 in Adults Aged 18 to 65 With Type 1 Myotonic Dystrophy

What this Trial Is About

Researchers are evaluating SRP-1003 in adults aged 18 to 65 with type 1 myotonic dystrophy DM1 in this phase 12a study. The trial aims to assess the safety, tolerability, how the drug moves through and affects the body pharmacokinetics and pharmacodynamics, comparing different doses of SRP-1003 to a placebo. Participants must have genetically confirmed DM1 with symptoms starting after age 12 and show clinical signs including myotonia. Participants will be randomly assigned to receive either SRP-1003 or a placebo through intravenous IV infusion or subcutaneous SC injection. The study has two parts Part 1 involves single doses, and Part 2 involves multiple doses. The treatment will be given under close monitoring to evaluate how the drug is processed and its effects. During the study, participants will have assessments including monitoring for adverse events up to 90 days for single-dose and 180 days for multiple-dose phases. Researchers will measure drug levels in the blood, changes in motor function tests such as hand opening time, timed walking tests, muscle strength, and quality of life scales specific to DM1. Safety and tolerability will be carefully observed throughout the study period that lasts several months.

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