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).
Official Title
A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
Keywords
Myotonic Dystrophy 1, Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Muscular Diseases, Steinert Disease, Inborn Genetic Diseases, Atrophic Muscular Disorders, Nervous System Heredodegenerative Disorders, Counterfeit Drugs, PGN-EDODM1 for infusion, PGN-EDODM1