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A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

NCT06833931 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL

Last updated 2025-06-24

No results posted yet for this study

Summary

The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).

The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).

Conditions

  • Duchenne Muscular Dystrophy (DMD)

Interventions

DRUG

IV infusion

IV infusion

OTHER

Placebo

IV infusion

Sponsors & Collaborators

  • PepGen Inc

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Max Age
16 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-17
Primary Completion
2025-05-28
Completion
2025-05-28
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06833931 on ClinicalTrials.gov