A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Phase 2

Suspended

• Conditions: Duchenne Muscular Dystrophy (DMD)
• Interventions: Drug: IV infusion; Other: Placebo

• Registration Number: NCT06833931
• Lead Sponsor: PepGen Inc

Brief Summary

The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).

The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).

Detailed Description

Not available

Study Timeline

• Study Start: 2024-12-17
• Study First Submitted: 2025-02-13
• Study First Submitted QC: 2025-02-13
• Study First Posted: 2025-02-19
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-13
• Last Update Posted: 2025-03-18
• Primary Completion: 2028-04-30
• Study Completion: 2030-04-30

Recruitment & Eligibility

• Status: SUSPENDED
• Sex: Male
• Target Recruitment: 16

Inclusion Criteria

• Confirmed diagnosis of DMD with a genetic alteration that can be treated by skipping exon 51
• Body weight at least 25kg (55lbs)
• Performance of Upper Limb (PUL) 2.0 entry score of at least 4

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks	IV infusion	-
PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks	Placebo	-
PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks	IV infusion	-
PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks	Placebo	-

Primary Outcome Measures

Name	Time	Method
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period)	Signing of informed consent to Week 28
Dystrophin levels (MAD period)	Baseline to Week 28
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)	Signing of informed consent to Week 108

Secondary Outcome Measures

Name	Time	Method
Plasma pharmacokinetic (PK) parameters (MAD period)	Baseline to Week 28	Area under the curve for concentration time of PGN-EDO51
Skeletal muscle concentration of PGN-EDO51 (MAD period)	Baseline to Week 28	Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses
Plasma pharmacokinetic (PK) parameters (LTE period)	Baseline to Week 104	PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels

Trial Locations

Locations (2)

Leeds Teaching Hospital NHS Trust; 🇬🇧 Leeds, United Kingdom

Oxford Children's Hospital; 🇬🇧 Oxford, United Kingdom