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

Last updated: February 27, 2025
Sponsor: PepGen Inc
Overall Status: Active - Not Recruiting

Phase

2

Condition

Muscular Dystrophy

Treatment

PGN-EDO51

Clinical Study ID

NCT06079736
PGN-EDO51-102
  • Ages 6-16
  • Male

Study Summary

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.

Eligibility Criteria

Inclusion

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

Exclusion Criteria:

  • Known history or presence of any clinically significant conditions that mayinterfere 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 infectionrequiring IV antibiotics within 4 weeks prior to Screening

  • Recent surgery requiring anesthesia within 3 months prior to Screening or expectedsurgery requiring general anesthesia during the study

Study Design

Total Participants: 7
Treatment Group(s): 1
Primary Treatment: PGN-EDO51
Phase: 2
Study Start date:
January 08, 2024
Estimated Completion Date:
April 30, 2027

Connect with a study center

  • British Columbia Children's Hospital

    Vancouver, British Columbia V6H1G9
    Canada

    Site Not Available

  • Stan Cassidy Centre for Rehabilitation

    Fredericton, New Brunswick E3B0C7
    Canada

    Site Not Available

  • Children's Hospital of Eastern Ontario (CHEO)

    Ottawa, Ontario K1H8L1
    Canada

    Site Not Available

  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario M5G0A4
    Canada

    Site Not Available

  • CHU de Québec

    Québec, Quebec G1V4G2
    Canada

    Site Not Available

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