New drugs aim to slow muscle decline in duchenne muscular dystrophy
NCT ID NCT07682129
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Mar 2032
An estimate. End dates often move.
- Lead sponsor
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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
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4 to 20 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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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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Get notified about this 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.
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The places running it
17 sites in 5 countries. The list below names each one and where it is.
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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.
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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
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Alder Hey Children's NHS Foundation Trust
Liverpool, L122AP, United Kingdom
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Centre Hospitalier Régional de la Citadelle
Liège, 4000, Belgium
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Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, 00168, Italy
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Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, 20162, Italy
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Freeman Hospital
Newcastle upon Tyne, NE1 3BZ, United Kingdom
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Great Ormond Street Hospital for Children
London, WC1N 3JH, United Kingdom
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Hospital Sant Joan de Deu
Barcelona, 08950, Spain
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Hospital Universitario Vall d'Hebron
Barcelona, 08035, Spain
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IRCCS Ospedale San Raffaele
Milan, 20132, Italy
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Leeds General Infirmary
Leeds, LS1 3EX, United Kingdom
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Leids Universitair Medisch Centrum
Leiden, 2333 ZA, Netherlands
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Ospedale Pediatrico Bambino Gesu
Rome, 00165, Italy
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Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU, United Kingdom
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA, Netherlands
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UZ Leuven
Leuven, 3000, Belgium
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University Hospital Gent
Ghent, 9000, Belgium
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Three-Hour online class help families manage bladder and bowel issues in duchenne muscular dystrophy?
- Can a new drug help boys with duchenne muscular dystrophy build muscle protein?
- AI-Powered heart scans aim to predict deadly complications in muscular dystrophy
- Wearable sensors could bring duchenne monitoring home
- Could a popular Weight-Loss drug help—or Harm—Those with duchenne muscular dystrophy?
- New drug could help boys with duchenne walk and move longer