PepGen - CONNECT2-EDO51

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDO51 with a Long-Term Extension in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

Hub Summary

The CONNECT2-EDO51 Phase 2 clinical trial is a multinational, randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) study, that will enroll ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. Participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. Participants will provide a muscle biopsy at baseline and then at week 25. The trial will evaluate the safety and tolerability of PGN-EDO51 and the levels of dystrophin in skeletal muscle following repeat dosing. All participants will have the opportunity to participate in an open-label extension for 108 weeks after completing the MAD period where all participants will receive only the investigational study drug PGN-EDO51.

Study Number: EU Clinical Trial Number: 2023-508383-29

Description by PepGen

The CONNECT2-EDO51 Phase 2 clinical trial is a multinational, randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) study, that will enrol ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. Participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. Participants will provide a muscle biopsy at baseline and then at week 25. The trial will evaluate the safety and tolerability of PGN-EDO51 and the levels of dystrophin in skeletal muscle following repeat dosing. All participants will have the opportunity to participate in an open-label extension for 108 weeks after completing the MAD period where all participants will receive the investigational study drug PGN-EDO51.

PGN-EDO51 is an investigational exon skipping drug designed to help cells “skip” over a section (called exon 51) of the transcript that codes for dystrophin. This could allow the body to create a shortened, but functional dystrophin protein. Dystrophin can potentially stabilize or even improve muscle function.
For the first 6 months of the study, participants will be randomly assigned to receive monthly IV infusions (through a needle in a vein) of either PGN-EDO51 or placebo. The placebo will look like PGN-EDO51 but does not contain any active investigational drug.

Participants will have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during these 6 months. During the rest of the study (about 2 years), participants will receive PGN-EDO51 only (no placebo).

Participation in this clinical research study is free, and all study-related costs will be covered. You will be compensated for your study visit time and will be eligible for reasonable travel expense reimbursement.

 
Primary Outcome Measures

• To evaluate the safety and tolerability of PGN-EDO51 following multiple doses in male participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.  
• To evaluate the levels of dystrophin in skeletal muscles following multiple doses of PGN EDO51.

 
Secondary Outcome Measures

• To evaluate what PGN-EDO51 does to the body and what the body does to PGN-EDO51 in plasma and muscle.

 
Can I take part?

 
Inclusion Criteria

 
Exclusion Criteria