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Exon Skipping Agent

PGN-EDO51 for Duchenne Muscular Dystrophy (CONNECT1-EDO51 Trial)

Phase 2
Recruiting
Research Sponsored by PepGen Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
Performance of Upper Limb (PUL) 2.0 entry score of at least 3 at Screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to week 108
Awards & highlights

Summary

This trial is being conducted to assess the safety and tolerability of giving multiple increasing doses of PGN-EDO51 through an IV to individuals with Duchenne muscular dystrophy. The trial will have

Who is the study for?
This trial is for males born with Duchenne muscular dystrophy (DMD) who are at least 10 years old, weigh a minimum of 25kg, and have a type that can be treated by skipping Exon 51. They should also be able to perform certain upper limb movements.
What is being tested?
The study tests the safety and effects of multiple IV doses of PGN-EDO51 in DMD patients. It includes up to 45 days of screening followed by a treatment and observation period lasting 16 weeks.
What are the potential side effects?
While specific side effects aren't listed here, participants will be monitored for any adverse reactions to PGN-EDO51 during their intravenous treatments over the course of the study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My DMD can potentially be treated by skipping Exon 51.
Select...
My upper limb function score is 3 or more.
Select...
My body weight is at least 25kg.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to week 108
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 108 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
Secondary study objectives
Dystrophin Levels (MAD period)
PK Plasma levels (LTE period)
Plasma pharmacokinetic (PK) parameters (MAD period)
+1 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: PGN-EDO51 at Dose Level 3 every 4 weeksExperimental Treatment1 Intervention
Group II: PGN-EDO51 at Dose Level 2 every 4 weeksExperimental Treatment1 Intervention
Group III: PGN-EDO51 at Dose Level 1 every 4 weeksExperimental Treatment1 Intervention

Find a Location

Who is running the clinical trial?

PepGen IncLead Sponsor
1 Previous Clinical Trials
24 Total Patients Enrolled
~4 spots leftby Mar 2025
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