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: 0
• Participant gender: Male

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: PepGen Inc

Criteria

Inclusion Criteria:

• Males by birth, age at least 8 years at the time of consent/assent provided

• Confirmed diagnosis of DMD able to be corrected by skipping Exon 51

• Body weight at least 25kg at Screening

• Performance of Upper Limb (PUL) 2.0 entry score of at least 3 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)

Exclusion Criteria:

• Known history or presence of any clinically significant conditions that may interfere with study safety assessments

• Treatment with any gene replacement therapy for the treatment of DMD at any time

• Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening

• Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study