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Exon Skipping Agent

PGN-EDO51 at Dose Level 1 every 4 weeks 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

CONNECT1-EDO51 Trial 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.Check my eligibility

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.See study design

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.

CONNECT1-EDO51 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My DMD can potentially be treated by skipping Exon 51.

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My upper limb function score is 3 or more.

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My body weight is at least 25kg.

CONNECT1-EDO51 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 108

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

Dystrophin Levels (MAD period)

PK Plasma levels (LTE period)

Plasma pharmacokinetic (PK) parameters (MAD period)

+1 more

CONNECT1-EDO51 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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

PepGen IncLead Sponsor

1 Previous Clinical Trials

24 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available positions for patients to participate in this clinical trial?

"Indeed, the details on clinicaltrials.gov indicate that this particular clinical trial is presently in the process of recruiting eligible candidates. The initial posting of the trial occurred on January 8th, 2024 and it was most recently updated on January 19th, 2024. This study aims to enroll a total of ten participants distributed among four different sites."

Answered by AI

What is the current number of individuals participating in this medical study?

"Indeed, clinicaltrials.gov verifies that this clinical trial is actively seeking individuals to participate. The initial posting of the study was on January 8th, 2024 and it was last updated on January 19th, 2024. The research team aims to recruit a total of 10 patients from four distinct locations."

Answered by AI

In the United States, are there multiple locations conducting this clinical investigation?

"This clinical trial is currently being conducted at 4 sites, including the Stan Cassidy Centre for Rehabilitation in Fredericton, Children's Hospital of Eastern Ontario (CHEO) in Ottawa, and The Hospital for Sick Children (SickKids) in Toronto. Additionally, there are 4 more locations participating in this study."

Answered by AI

Has PGN-EDO51 at Dose Level 1, administered every four weeks, received approval from the FDA?

"Based on our assessment at Power, the safety rating for PGN-EDO51 administered at Dose Level 1 every 4 weeks is a 2. This evaluation considers that it is a Phase 2 trial with available data supporting its safety but lacking evidence of efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06079736.

All > Muscular Dystrophy