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New hope for DM1: Long-Term safety trial of PGN-EDODM1 now recruiting

NCT ID NCT07220603

Recruiting now Disease control Sponsor: PepGen Inc Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is testing the long-term safety of an experimental drug called PGN-EDODM1 in 48 adults with myotonic dystrophy type 1 (DM1) who have already taken the drug in a previous study. Participants will receive the drug by IV infusion and be monitored for side effects. The goal is to see if the drug remains safe and tolerable over a longer period.

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 (given by IV infusion)
What this could lead to
If successful, this study could support the continued development of PGN-EDODM1 as a treatment to control myotonic dystrophy type 1 symptoms.
What could go wrong
This is an early-phase, open-label extension study with no placebo group, so results are preliminary. The drug may still fail in larger trials or cause unexpected side effects.

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 musculoskeletal system disorder myotonic dystrophy myotonic dystrophy type 1 myotonic syndrome nervous system disorder 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • CIUSSS du Saguenay-Lac-Saint-Jean

    RECRUITING

    Saguenay, Canada

    Contact Email: •••••@•••••

  • Ottawa Hospital Research Institute

    RECRUITING

    Ottawa, Ontario, Canada

    Contact Email: •••••@•••••

  • University of Calgary

    RECRUITING

    Calgary, Alberta, T2N 4Z6, Canada

    Contact Email: •••••@•••••

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