Tracking the future: Long-Term study for duchenne gene therapy patients

NCT ID NCT05967351

Summary

This study is tracking the long-term safety and effectiveness of a gene therapy called delandistrogene moxeparvovec (SRP-9001) in people with Duchenne muscular dystrophy. It will follow 400 participants who have already received the therapy in a previous clinical trial. The goal is to see how well the treatment continues to work and monitor for any long-term side effects over time.

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Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Chiildren's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Health Specialty Center

    Dallas, Texas, 75207, United States

  • Children's Hospital Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045-7106, United States

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23510, United States

  • Columbia University Pediatric Neuromuscular Center

    New York, New York, 10032, United States

  • Great Ormond Street Hospital For Children NHS Foundation Trust

    London, Greater London, WC1N 1EJ, United Kingdom

  • Hong Kong Children's Hospital

    Hong Kong, Hong Kong

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat, Barcelona, 08950, Spain

  • Hospital Universitario y Politecnico La fe. Neurology Department

    Valencia, 46026, Spain

  • Institute of Translational and Clinical Research

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • Kobe University Hospital

    Kobe, Hyōgo, 650-0017, Japan

  • Lenox Baker Children's Hospital (Duke University)

    Durham, North Carolina, 27705, United States

  • Lucile Packard Children's Hospital Stanford (LPCH)

    Palo Alto, California, 94304, United States

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo, 187-8551, Japan

  • National Taiwan University Hospital

    Taipei, 100225, Taiwan

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, Oxfordshire, OX3 9DU, United Kingdom

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • The Johns Hopkins Hospital, Rubenstein Child Health Bldg

    Baltimore, Maryland, 21287, United States

  • Tokyo Women's Medical University Hospital

    Tokyo, 1628666, Japan

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico

    Genova, 16147, Italy

  • UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore

    Roma, 00168, Italy

  • Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)

    Ghent, East Flanders, 9000, Belgium

  • University of California, Davis

    Sacramento, California, 95817, United States

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32610, United States

  • University of Iowa ICTS Clinical Research Unit

    Iowa City, Iowa, 52242, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah Health

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Essen - Klinik für Kinderheilkunde I

    Essen, North Rhine-Westphalia, 45147, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.