New drug PGN-EDODM1 tested for muscle disease

NCT ID NCT06204809

Completed Disease control Sponsor: PepGen Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tested a single dose of the drug PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a genetic muscle disorder. The main goal was to check safety and how the body handles the drug. The study is complete, but results are not yet available.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: PGN-EDODM1
What this could lead to: If safe, this could pave the way for a treatment that targets the root cause of myotonic dystrophy type 1.
What could go wrong: This is an early Phase 1 safety trial with only 24 people. It does not yet test if the drug works, and side effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

hereditary disease inherited neurodegenerative disorder muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder myotonic dystrophy myotonic dystrophy type 1 myotonic syndrome nervous system disorder neurodegenerative disease neuromuscular disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MYOTONIC DYSTROPHY 1

Contacts and locations

Locations

CIUSSS du Saguenay-Lac-Saint-Jean

Chicoutimi, Quebec, Canada
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Ottawa Hospital Research Institute (OHRI)

Ottawa, Ontario, Canada
Rare Disease Research

Atlanta, Georgia, 30329, United States
Salford Royal Hospital

Salford, United Kingdom
Stanford University

Palo Alto, California, 94304, United States
UCI Center for Clinical Research

Irvine, California, 92697, United States
University College London Hospital

London, UK, NW1 2PG, United Kingdom
University of Calgary

Calgary, Alberta, T3M 1M4, Canada
University of Kansas Medical Center

Fairway, Kansas, 66205, United States
University of Rochester Medical Center

Rochester, New York, 14642, United States
Virginia Commonwealth University

Richmond, Virginia, 23298, United States

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