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New drug trial for duchenne MD halted early – what we know

NCT ID NCT06079736

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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 phase 2 study tested a drug called PGN-EDO51 in 7 people with Duchenne muscular dystrophy whose genetic mutation can be corrected by skipping exon 51. The drug was given by IV infusion to see if it is safe and tolerable. The trial was terminated, so results are limited.

What this could mean

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

Active substance
PGN-EDO51 (a drug given by IV infusion to help produce a shorter but functional dystrophin protein)
What this could lead to
If successful, this could lead to a treatment that slows muscle decline in Duchenne muscular dystrophy patients who are candidates for exon 51 skipping.
What could go wrong
This is an early-phase, small trial (7 participants) focused on safety, not yet on effectiveness. The study was terminated, which raises uncertainty about its future.

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

7 people

The number who actually took part.

Started

Jan 2024

Finished

Aug 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

6 to 16 years

Sex

Male participants only

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 DMD able to be corrected by skipping Exon 51 * Body weight at least 18kg at Screening * Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD) Exclusion Criteria: * Known history or presence of any clinically significant conditions that may interfere with study safety assessments * Treatment with any gene replacement therapy for the treatment of DMD at any time * Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening * Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy muscular dystrophy neuromuscular disease

As listed by the trial registrant

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

Duchenne muscular dystrophy

Contacts and locations

Locations

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H1G9, Canada

  • CHU de Québec

    Québec, Quebec, G1V4G2, Canada

  • Children's Hospital of Eastern Ontario (CHEO)

    Ottawa, Ontario, K1H8L1, Canada

  • Stan Cassidy Centre for Rehabilitation

    Fredericton, New Brunswick, E3B0C7, Canada

  • The Hospital for Sick Children (SickKids)

    Toronto, Ontario, M5G0A4, Canada

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