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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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

Locations

  • Alder Hey Children's NHS Foundation Trust

    NOT_YET_RECRUITING

    Liverpool, L122AP, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Centre Hospitalier Régional de la Citadelle

    RECRUITING

    Liège, 4000, Belgium

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    RECRUITING

    Milan, 20162, Italy

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Freeman Hospital

    RECRUITING

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Great Ormond Street Hospital for Children

    RECRUITING

    London, WC1N 3JH, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Hospital Sant Joan de Deu

    RECRUITING

    Barcelona, 08950, Spain

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • IRCCS Ospedale San Raffaele

    RECRUITING

    Milan, 20132, Italy

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Leeds General Infirmary

    RECRUITING

    Leeds, LS1 3EX, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Ospedale Pediatrico Bambino Gesu

    RECRUITING

    Rome, 00165, Italy

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Oxford University Hospitals NHS Foundation Trust

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Royal Manchester Children's Hospital

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University Hospital Gent

    RECRUITING

    Ghent, 9000, Belgium

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.