Actively Recruiting
Phase 2 Study of PGN-EDODM1 Treatment in Adults with Myotonic Dystrophy Type 1 to Assess Safety and Tolerability
Led by PepGen Inc · Updated on 2026-07-29
24
Participants Needed
12
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of an investigational medicine called PGN-EDODM1 to understand how safe and tolerable multiple doses are for adults with myotonic dystrophy type 1 DM1. This phase 2 study compares PGN-EDODM1 to a placebo to assess its safety and tolerability in people with this condition. Participants will be randomly assigned to receive either PGN-EDODM1 or a placebo. The treatment is given by intravenous infusion once every 4 weeks for a total of 12 weeks. Those in the PGN-EDODM1 group will receive ascending doses, while the placebo group will receive saline infusions on the same schedule. During the study, participants will be monitored for safety by tracking adverse events from the start through Day 112. Researchers will measure drug levels in the blood, changes in muscle tissue, myotonia severity, hand grip strength, and mobility using specific tests. The total participation lasts through about 16 weeks, including treatment and follow-up assessments.
CONDITIONS
Brief Title
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Research Team
P
PepGen
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
