Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for duchenne: experimental drug targets genetic flaw

NCT ID NCT07037862

Disease control Sponsor: Entrada Therapeutics, 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Aug 14, 2026 · Updated 3 times

Summary

This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effective dose, and second, to check how well it works. Participants receive multiple IV infusions and are monitored with blood tests, muscle biopsies, and physical exams.

What this could mean

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

Active substance
ENTR-601-44 (a drug designed to help muscle cells produce a shortened but functional dystrophin protein)
What this could lead to
If successful, this could lead to a treatment that slows muscle decline and improves function in people with Duchenne muscular dystrophy who are eligible for exon 44 skipping.
What could go wrong
This is an early-phase trial with only 24 participants, so results may not apply to everyone. The drug may cause side effects or fail to show meaningful benefit.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 24 people

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

Started

Jun 2025

Expected to finish

Mar 2029

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

4 to 20 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.

Principal inclusion criteria 1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor. 2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator. 3. Part A: 4-20 years of age, inclusive. 4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening 5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator. 6. Other protocol-defined criteria apply. Principal exclusion criteria 1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements. 2. Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety. 3. Use of the following medications: 1. Prior treatment with any exon skipping therapy at any time 2. Prior treatment with any gene therapy at any time 3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents 4. Use of an immunosuppressants (other than oral corticosteroids for DMD conditions) 5. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat 4. Laboratory abnormalities. 5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy. 6. Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) \>450 msec at Screening or prior to the first dose of study drug on Day 1. 7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer). 8. Other protocol-defined criteria apply.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Duchenne muscular dystrophy (DMD) are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

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

Duchenne muscular dystrophy (DMD)

How to take part

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

  1. The places running it

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

  2. 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 ↗

  3. 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

Locations

  • Alder Hey Children's NHS Foundation Trust

    RECRUITING

    Liverpool, L122AP, United Kingdom

  • Centre Hospitalier Régional de la Citadelle

    RECRUITING

    Liège, 4000, Belgium

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    RECRUITING

    Milan, 20162, Italy

  • Freeman Hospital

    RECRUITING

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Great Ormond Street Hospital for Children

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • IRCCS Ospedale San Raffaele

    RECRUITING

    Milan, 20132, Italy

  • Leeds General Infirmary

    RECRUITING

    Leeds, LS1 3EX, United Kingdom

  • Ospedale Pediatrico Bambino Gesu

    RECRUITING

    Rome, 00165, Italy

  • Oxford University Hospitals NHS Foundation Trust

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • Royal Manchester Children's Hospital

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • University Hospital Gent

    RECRUITING

    Ghent, 9000, Belgium

More trials for these conditions

Other studies related to the condition(s) this trial covers.