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New drug PGN-EDODM1 tested for muscle disease

NCT ID NCT06204809

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

24 people

The number who actually took part.

Started

Dec 2023

Finished

Oct 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 60 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 Exclusion Criteria: * 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.

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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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