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New drugs aim to slow muscle decline in duchenne muscular dystrophy

NCT ID NCT07682129

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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 02, 2026 · Last updated Jul 02, 2026

Summary

This study tests two experimental drugs, ENTR-601-44 and ENTR-601-45, in people with Duchenne muscular dystrophy (DMD) who have already taken part in earlier studies. The goal is to see if long-term use is safe and tolerable, and whether it can help maintain muscle function. Participants receive regular intravenous infusions and undergo tests like blood work, physical exams, and muscle biopsies. The study also tracks changes in walking speed and other physical abilities over time.

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 and ENTR-601-45 (intravenous infusions)
What this could lead to
If successful, this could show that long-term treatment with these drugs is safe and may help preserve muscle function in people with Duchenne muscular dystrophy.
What could go wrong
This is an early-phase extension study, so it is small and not designed to prove effectiveness. The drugs may cause side effects or not work as hoped.

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 80 people

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

Expected to start

Aug 2026

An estimate. Start dates often move.

Expected to finish

Mar 2032

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.

Inclusion Criteria: * Willing and able to provide consent (if at age of majority) or assent (if a minor) * Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201 * Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual intercourse Exclusion Criteria: * Any change from the applicable parent study eligibility criteria, including safety events during the parent study, that in the opinion of the investigator in consultation with the medical monitor and/or sponsor designee precludes safe use of study drug * Participant has a condition or circumstance that in the view of the investigator places the subject at high risk of poor treatment compliance or for not completing the study

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

How to take part

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

  1. The places running it

    17 sites in 5 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    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

    Liverpool, L122AP, United Kingdom

  • Centre Hospitalier Régional de la Citadelle

    Liège, 4000, Belgium

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Roma, 00168, Italy

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    Milan, 20162, Italy

  • Freeman Hospital

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Great Ormond Street Hospital for Children

    London, WC1N 3JH, United Kingdom

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035, Spain

  • IRCCS Ospedale San Raffaele

    Milan, 20132, Italy

  • Leeds General Infirmary

    Leeds, LS1 3EX, United Kingdom

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA, Netherlands

  • Ospedale Pediatrico Bambino Gesu

    Rome, 00165, Italy

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU, United Kingdom

  • Royal Manchester Children's Hospital

    Manchester, M13 9WL, United Kingdom

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA, Netherlands

  • UZ Leuven

    Leuven, 3000, Belgium

  • University Hospital Gent

    Ghent, 9000, Belgium

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