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

Completed · Phase 1 · Has a placebo group

Conditions studied: Myotonic Dystrophy 1

In brief

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).

Key facts

Study ID

NCT06204809

Run by

PepGen Inc

People needed

24

Starts

2023-12-12

Expected to finish

2025-10-28

Last updated by the study team

2026-02-13

Who can join

Age: 18 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with 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 at least moderate pressure from the examiner)
• Presence of myotonia

You may not qualify if…

• Congenital DM1
• Known history or presence of any clinically significant conditions that may interfere 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.

Where it is running

• UCI Center for Clinical Research — Irvine, California, United States
• Stanford University — Palo Alto, California, United States
• Rare Disease Research — Atlanta, Georgia, United States
• University of Kansas Medical Center — Fairway, Kansas, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Rochester Medical Center — Rochester, New York, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• University of Calgary — Calgary, Alberta, Canada
• Ottawa Hospital Research Institute (OHRI) — Ottawa, Ontario, Canada
• CIUSSS du Saguenay-Lac-Saint-Jean — Chicoutimi, Quebec, Canada
• University College London Hospital — London, UK, United Kingdom
• Salford Royal Hospital — Salford, United Kingdom

Full record on ClinicalTrials.gov

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