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New hope for muscle stiffness: experimental drug PGN-EDODM1 enters Mid-Stage trial

NCT ID NCT06667453

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jul 30, 2026 · Updated 3 times

Summary

This study tests an investigational drug called PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a condition causing muscle stiffness and weakness. Participants receive multiple doses of the drug or a placebo by IV to check safety and how the body processes it. The goal is to see if the drug is safe and tolerable.

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 successful, this could point toward a treatment that controls symptoms of myotonic dystrophy type 1, like muscle stiffness and weakness.
What could go wrong
This is an early Phase 2 trial with only 24 participants, so results may not apply to everyone. The drug is still experimental and may not work or could have side effects.
Why investors are watching

PepGen is running a small phase 2 trial of its drug PGN-EDODM1 in 24 adults with myotonic dystrophy type 1, a rare genetic muscle disease. For a micro-cap company with few other products, this readout is a key test of whether the drug is safe and tolerable, and whether it shows enough promise to justify further development.

If it works: A positive result could show that PGN-EDODM1 is safe and well-tolerated, which would support PepGen moving the drug into larger trials. That progress could strengthen the company's position with partners or investors.

If it fails: A failure or delay could set back the drug's development and hurt PepGen's prospects, since the company's value depends heavily on this program. Trials at this stage often fail, so a negative outcome is a real possibility.

AI-written from the trial record. Speculative, and not investment advice.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2024

Expected to finish

Mar 2027

An estimate. End dates often move.

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

16 to 65 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 * Presence of myotonia * Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed * Body Mass Index (BMI) of \< 35.0 kg/m\^2 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 considered clinically significant by the Investigator * Medications specific for the treatment of myotonia within 2 weeks prior to screening * Percent predicted forced vital capacity (FVC) \<40% * Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening Note: Other inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

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

Myotonic dystrophy 1

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites in 4 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • CIUSSS du Saguenay-Lac-Saint-Jean

    RECRUITING

    Chicoutimi, Quebec, Canada

  • Cardiff and Vale Hospital

    RECRUITING

    Cardiff, United Kingdom

    Contact Email: •••••@•••••

  • Kyungpook National University Chilgok Hospital

    RECRUITING

    Daegu, South Korea

  • Montreal Neurological Institute

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

    Contact Email: •••••@•••••

  • Newcastle Upon Tyne Hospitals

    RECRUITING

    Newcastle upon Tyne, NE7 7DN, United Kingdom

    Contact Email: •••••@•••••

  • Ottawa Hospital Research Institute (OHRI)

    RECRUITING

    Ottawa, Ontario, Canada

  • Pacific Clinical Research Network Auckland

    RECRUITING

    Takapuna, Auckland, 0622, New Zealand

  • Pusan National University Yangsan Hospital

    RECRUITING

    Pusan, Gyeongsangnam-do, South Korea

    Contact Email: •••••@•••••

  • Salford Royal Hospital

    RECRUITING

    Salford, England, United Kingdom

    Contact Email: •••••@•••••

  • Seoul National University Hospital

    RECRUITING

    Seoul, South Korea

  • University College London Hospitals NHS Foundation Trust

    RECRUITING

    London, UK, United Kingdom

    Contact Email: •••••@•••••

  • University of Calgary

    RECRUITING

    Calgary, Alberta, T2N 4Z6, Canada

    Contact Email: •••••@•••••

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