Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1

Last updated: March 13, 2025
Sponsor: PepGen Inc
Overall Status: Active - Recruiting

Phase

1

Condition

Muscular Dystrophy

Treatment

PGN-EDODM1 for infusion

PGN-EDODM1

Placebo

Clinical Study ID

NCT06204809
PGN-EDODM1-101
  • Ages 18-50
  • All Genders

Study Summary

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK genewith at least 100 CTG repeats

  • Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against atleast moderate pressure from the examiner)

  • Presence of myotonia

Exclusion

Exclusion Criteria:

  • Congenital DM1

  • Known history or presence of any clinically significant conditions that mayinterfere with study safety assessments

  • Abnormal laboratory tests at screening

  • Medications specific for the treatment of myotonia within 2 weeks prior to screening

  • Percent predicted forced vital capacity (FVC) <40%

Note: Other inclusion and exclusion criteria may apply.

Study Design

Total Participants: 32
Treatment Group(s): 3
Primary Treatment: PGN-EDODM1 for infusion
Phase: 1
Study Start date:
December 12, 2023
Estimated Completion Date:
April 30, 2025

Connect with a study center

  • University of Calgary

    Calgary, Alberta T3M 1M4
    Canada

    Active - Recruiting

  • Ottawa Hospital Research Institute (OHRI)

    Ottawa, Ontario
    Canada

    Active - Recruiting

  • CIUSSS du Saguenay-Lac-Saint-Jean

    Chicoutimi, Quebec
    Canada

    Active - Recruiting

  • University College London Hospital

    London, UK NW1 2PG
    United Kingdom

    Active - Recruiting

  • Salford Royal Hospital

    Salford,
    United Kingdom

    Active - Recruiting

  • UCI Center for Clinical Research

    Irvine, California 92697
    United States

    Active - Recruiting

  • Stanford University

    Palo Alto, California 94304
    United States

    Active - Recruiting

  • Rare Disease Research

    Atlanta, Georgia 30329
    United States

    Active - Recruiting

  • Rare Disease Research, LLC

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • University of Kansas Medical Center

    Fairway, Kansas 66205
    United States

    Active - Recruiting

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.