New drug hopes to slow duchenne muscle damage in kids and young adults
NCT ID NCT07037862
First seen Nov 21, 2025 · Last updated May 07, 2026 · Updated 18 times
Summary
This study tests an investigational medicine called ENTR-601-44 for people with Duchenne muscular dystrophy (DMD) whose genetic mutation allows skipping of exon 44. The trial has two parts: first to find the safest dose, then to see if it improves muscle function compared to a placebo. About 24 participants, aged 4 to 20 and able to walk, will receive IV infusions and undergo regular checkups, blood tests, and muscle biopsies.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Alder Hey Children's NHS Foundation Trust
NOT_YET_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
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Conditions
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