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

NCT06079736 ↗

Phase 2 Terminated 7 enrolled PepGen Inc
Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2024-01-03
Primary completion
2025-08-28
Completion
2025-08-28

Outcome

Missed primary endpoint

Stopped: “PGN-EDO51 development terminated by Sponsor”

release “PGN-EDO51 did not achieve target dystrophin levels in CONNECT1-EDO51 trial” pepgen.com ↗

Drugs

EvaluationDrugModalityDoseRoute
Subject PGN-EDO51 Unknown — Intravenous

Indications

Duchenne muscular dystrophy