A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
Status:
Recruiting
Trial end date:
2027-05-01
Target enrollment:
Participant gender:
Summary
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple
ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne
muscular dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term
safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and
Observation Period (16 weeks), and an Extension Period (108 weeks).