A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)

What this Trial Is About

Researchers are evaluating the safety and tolerability of multiple doses of an investigational medicine called PGN-EDODM1 in adults with myotonic dystrophy type 1 (DM1). This randomized, double-blind study compares PGN-EDODM1 to a placebo to better understand its effects on this condition. The study is a Phase 2 trial sponsored by PepGen Inc and aims to assess how well participants tolerate the treatment and monitor any adverse events. Participants will be randomly assigned to receive either ascending doses of PGN-EDODM1 or a placebo, both given by intravenous infusion once every 4 weeks for a total of 12 weeks. The treatment period spans these 12 weeks during which doses are administered and safety is closely monitored. The placebo group receives saline infusions on the same schedule to provide comparison data. During the study, participants will be regularly assessed for safety by tracking any adverse events from the start through day 112. Researchers will also measure how the drug behaves in the body, including plasma concentration and half-life, as well as changes in muscle function and myotonia using video hand opening time, hand grip strength, and 10 meter walk/run tests. Muscle biopsies will be performed to study tissue changes. The entire participation period includes baseline assessments, treatment, and follow-up evaluations lasting up to about 112 days.

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1