A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2024
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).
Epistemonikos ID: e25b9af5b3a7bc8d33008b1b503b88d688ce465f
First added on: May 15, 2024