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

Phase 2

Recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: PGN-EDO51

• Registration Number: NCT06079736
• Lead Sponsor: PepGen Inc

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-09-29
• Study First Submitted QC: 2023-10-05
• Study First Posted: 2023-10-12
• Study Start: 2024-01-08
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-03
• Last Update Posted: 2025-01-07
• Primary Completion: 2025-11
• Study Completion: 2027-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 11

Inclusion Criteria

• Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
• Body weight at least 18kg at Screening
• Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
PGN-EDO51 at Dose Level 1 every 4 weeks	PGN-EDO51	-
PGN-EDO51 at Dose Level 2 every 4 weeks	PGN-EDO51	-
PGN-EDO51 at Dose Level 3 every 4 weeks	PGN-EDO51	-

Primary Outcome Measures

Name	Time	Method
Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)	Baseline to Week 108	Adverse events and serious adverse events
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)	Baseline to Week 16	Adverse events and serious adverse events

Secondary Outcome Measures

Name	Time	Method
Plasma pharmacokinetic (PK) parameters (MAD period)	Baseline to Week 12	Area under the curve for concentration time of PGN-EDO51
PK Plasma levels (LTE period)	Baseline to Week 104	PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels
Skeletal muscle concentration of PGN-EDO51 (MAD period)	Baseline to Week 16	Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses
Dystrophin Levels (MAD period)	Baseline to Week 16	Change from baseline in dystrophin levels measured after multiple doses

Trial Locations

Locations (5)

British Columbia Children's Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Stan Cassidy Centre for Rehabilitation; 🇨🇦 Fredericton, New Brunswick, Canada

Children's Hospital of Eastern Ontario (CHEO); 🇨🇦 Ottawa, Ontario, Canada

The Hospital for Sick Children (SickKids); 🇨🇦 Toronto, Ontario, Canada

CHU de Québec; 🇨🇦 Québec, Quebec, Canada