A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

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)

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.

Study Overview

• Status: Terminated
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Drug: PGN-EDO51

• Study Type: Interventional
• Enrollment (Actual): 7
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H1G9
      • British Columbia Children's Hospital
  • New Brunswick
    • Fredericton, New Brunswick, Canada, E3B0C7
      • Stan Cassidy Centre for Rehabilitation
  • Ontario
    • Ottawa, Ontario, Canada, K1H8L1
      • Children's Hospital of Eastern Ontario (CHEO)
    • Toronto, Ontario, Canada, M5G0A4
      • The Hospital for Sick Children (SickKids)
  • Quebec
    • Québec, Quebec, Canada, G1V4G2
      • Chu De Quebec

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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)

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PGN-EDO51 at Dose Level 1 every 4 weeks	Drug: PGN-EDO51; IV infusion
Experimental: PGN-EDO51 at Dose Level 2 every 4 weeks	Drug: PGN-EDO51; IV infusion

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma pharmacokinetic (PK) parameters (MAD period)	Maximum observed plasma concentration of PGN-EDO51	Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)	Apparent terminal half-life of PGN-EDO51	Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)	Area under the curve for concentration time of PGN-EDO51	Baseline to Week 12
PK Plasma levels (LTE period)	PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels	Baseline to Week 104
Plasma pharmacokinetic (PK) parameters (MAD period)	Time to maximum observed plasma concentration of PGN-EDO51	Baseline to Week 12
Skeletal muscle concentration of PGN-EDO51 (MAD period)	Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses	Baseline to Week 16
Dystrophin Levels (MAD period)	Change from baseline in dystrophin levels measured after multiple doses	Baseline to Week 16

Collaborators and Investigators

• Sponsor: PepGen Inc

Study record dates

Study Major Dates

• Study Start (Actual): January 3, 2024
• Primary Completion (Actual): August 28, 2025
• Study Completion (Actual): August 28, 2025

Study Registration Dates

• First Submitted: September 29, 2023
• First Submitted That Met QC Criteria: October 5, 2023
• First Posted (Actual): October 12, 2023

Study Record Updates

• Last Update Posted (Actual): December 2, 2025
• Last Update Submitted That Met QC Criteria: December 1, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Neuromuscular Disease; Oligonucleotide; Antisense oligonucleotide; Muscular Dystrophies; Exon 51; Enhanced Delivery Oligonucleotide; Peptide-conjugated phosphorodiamidate; Morpholino oligomer; Next-generation oligonucleotide; Cell-penetrating peptide; Dystrophin production; Splice correcting oligonucleotide; Endosomal Escape; Delivery to the cell nucleus; phosphorodiamidate morpholino oligomer (PPMO)
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophies; Muscular Dystrophy, Duchenne; Neuromuscular Diseases
• Other Study ID Numbers: PGN-EDO51-102

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes