NCT06079736

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

A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: PepGen Inc · Industry
• Sex: Male
• Age: 6 Years – 16 Years
• Healthy volunteers: Not accepted

Summary

The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks). 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.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	PGN-EDO51	IV infusion

Timeline

• Start date: 2024-01-03
• Primary completion: 2025-08-28
• Completion: 2025-08-28
• First posted: 2023-10-12
• Last updated: 2025-12-02

Locations

5 sites across 1 country: Canada