New hope for duchenne: experimental drug targets genetic flaw
NCT ID NCT07037862
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.
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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.
Contacts and locations
Locations
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Alder Hey Children's NHS Foundation Trust
RECRUITINGLiverpool, L122AP, United Kingdom
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Centre Hospitalier Régional de la Citadelle
RECRUITINGLiège, 4000, Belgium
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Fondazione Serena Onlus - Centro Clinico NeMO Milano
RECRUITINGMilan, 20162, Italy
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Freeman Hospital
RECRUITINGNewcastle upon Tyne, NE1 3BZ, United Kingdom
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Great Ormond Street Hospital for Children
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
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Hospital Universitario Vall d'Hebron
RECRUITINGBarcelona, 08035, Spain
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IRCCS Ospedale San Raffaele
RECRUITINGMilan, 20132, Italy
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Leeds General Infirmary
RECRUITINGLeeds, LS1 3EX, United Kingdom
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Ospedale Pediatrico Bambino Gesu
RECRUITINGRome, 00165, Italy
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Oxford University Hospitals NHS Foundation Trust
RECRUITINGOxford, OX3 9DU, United Kingdom
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Royal Manchester Children's Hospital
NOT_YET_RECRUITINGManchester, M13 9WL, United Kingdom
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UZ Leuven
RECRUITINGLeuven, 3000, Belgium
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University Hospital Gent
RECRUITINGGhent, 9000, Belgium
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new drug help boys with duchenne muscular dystrophy build muscle protein?
- New drugs aim to slow muscle decline in duchenne muscular dystrophy
- 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