Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Summary

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Timeline

Start

2023-12-12

Primary completion

2025-10-28

Completion

2025-10-28

Drugs

Indications