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Race against time: High-Dose drug trial aims to keep boys walking longer

NCT ID NCT03992430

Ongoing Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

Summary

This study is testing whether higher doses of the drug eteplirsen are safe and more effective at slowing muscle decline in boys with Duchenne muscular dystrophy (DMD). It involves about 160 boys aged 4 and older who can still walk and have a specific genetic mutation. The main goal is to see if higher doses help maintain muscle function and delay the loss of walking ability over several years.

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

  • Akdeniz Universitesi Tip Fakultesi

    Antalya, 07059, Turkey (Türkiye)

  • All India Institute of Medical Sciences

    New Delhi, 110029, India

  • Aster RV Hospital

    Bengaluru, 560078, India

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Brno Klinika detske neurologie

    Brno, 613 00, Czechia

  • CHRU de Strasbourg

    Strasbourg, 67098, France

  • Charité Universitätsmedizin Berlin CVK

    Berlin, 13353, Germany

  • Children's Department and Department for Children's Habilitation at Stavanger University Hospital

    Stavanger, 4011, Norway

  • Children's Health Ireland (CHI) at Temple Street

    Dublin, D01 XD99, Ireland

  • Christian Medical College

    Vellore, 632004, India

  • Clinic for Neurology and Psychiatry for Children and Youth

    Belgrade, 11000, Serbia

  • Deenanath Mangeshkar Hospital & Research Centre

    Pune, 411004, India

  • Fakultni nemocnice v Motole

    Prague, 150 06, Czechia

  • Fondazione Policlinico Universitario A. Gemelli- IRCCS

    Rome, 00168, Italy

  • Hopital Armand Trousseau

    Paris, 75571, France

  • Hopital Femme Mere Enfant

    Bron, 69677, France

  • Hospital Pablo Tobón Uribe

    Medellín, 50034, Colombia

  • Hospital Sant Joan de Deu

    Barcelona, 8950, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Hospital Universitario San Ignacio

    Bogotá, 110231, Colombia

  • IASO Children's Hospital

    Marousi, Attica, 151 23, Greece

  • IRCCS Instituto Gianna Gaslini

    Genova, 16147, Italy

  • Instituto Neurologico de Colombia (INDEC)

    Medellín, 50012, Colombia

  • Instituto de Investigaciones Clinicas para la Salud A.C

    Durango, 34000, Mexico

  • Irbid Specialty Hospital

    Irbid, 22110, Jordan

  • Istiklal Hosptial (IST)

    Amman, 11196, Jordan

  • Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)

    Madurai, 626022, India

  • Kaohsiung Medical University

    Kaohsiung City, 80756, Taiwan

  • Klinika Neurologii Rozwojowej

    Gdansk, Pomeranian Voivodeship, 80-211, Poland

  • Kyungpook National University Chilgok Hospital

    Daegu, 41404, South Korea

  • Leeds Teaching Hospitals NHS Trust

    Leeds, West Yorkshire, LS1 3EX, United Kingdom

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZC, Netherlands

  • Mersin University Medical Faculty

    Mersin, 33110, Turkey (Türkiye)

  • Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"

    Belgrade, 190133, Serbia

  • National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"

    Bucharest, 41408, Romania

  • National Taiwan University Hospital

    Taipei, 10071, Taiwan

  • Neurociencias Estudios Clínicos S.C.

    Culiacán, Sinaloa, 80020, Mexico

  • New Zealand Clinical Research - Auckland

    Auckland, 1010, New Zealand

  • Nizam's Institute of Medical Sciences

    Hyderabad, 500082, India

  • Oslo Universitetssykehus HF Rikshospitalet

    Oslo, 0450, Norway

  • Pharmaceutical Research Center/Jordan University of Science and Technology

    Irbid, 22110, Jordan

  • Pusan National University Yangsan Hospital

    Yangsan, 50612, South Korea

  • Radboud University Nijmegen Medical Centre

    Nijmegen, 6525GA, Netherlands

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30318, United States

  • Rigshospitalet Copenhagen University Hospital

    Copenhagen, 2100, Denmark

  • Royal Institute of Child Neurosciences

    Ahmedabad, 380054, India

  • Samsung Medical Center

    Seoul, 6351, South Korea

  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

    Budapest, 1082, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Sir Ganga Ram Hospital

    New Delhi, 110060, India

  • The Specialty Hospital (TSH)/Advanced Clinical Center

    Amman, 11194, Jordan

  • UCL Institute of Child Health Great Ormond Street

    London, WC1N 1EH, United Kingdom

  • University Children's Hospital

    Belgrade, 11000, Serbia

  • University Medical Centre Ljubljana

    Ljubljana, 1000, Slovenia

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Florida

    Gainesville, Florida, 32610-3010, United States

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • Universitätsspital Basel

    Basel, 4031, Switzerland

Conditions

Explore the condition pages connected to this study.

MUSCULAR DYSTROPHY, DUCHENNE