New hope for duchenne: experimental drug ENTR-601-45 enters human trials
NCT ID NCT07038824
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an investigational drug called ENTR-601-45 in people with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. About 24 males aged 4 to 20 will receive the drug or a placebo through IV infusions, along with regular checkups and muscle biopsies.
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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
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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 Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
RECRUITINGRome, 00168, Italy
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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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Leids Universitair Medisch Centrum
RECRUITINGLeiden, 2333 ZA, Netherlands
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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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Stichting Radboud Universitair Medisch Centrum
RECRUITINGNijmegen, 6525 GA, Netherlands
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UZ Leuven
RECRUITINGLeuven, 3000, Belgium
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University Hospital Gent
RECRUITINGGhent, 9000, Belgium
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More trials for these conditions
Other studies related to the condition(s) this trial covers.
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