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Decade-Long watch: tracking gene Therapy's lasting impact on rare metabolic disease

NCT ID NCT06636383

Recruiting now Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

Summary

This study will monitor 140 people with Glycogen Storage Disease Type Ia (GSDIa) for at least 10 years after they received an investigational gene therapy called DTX401. The main goal is to understand the long-term safety and effectiveness of the treatment by tracking health outcomes and daily management of the disease. It is an observational study, meaning researchers will collect data but not administer any new treatment.

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

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Children's Hospital Colorado

    RECRUITING

    Denver, Colorado, 80045, United States

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Fujita Health University Hospital

    RECRUITING

    Toyoake, 470-1192, Japan

  • Groningen University

    RECRUITING

    Groningen, Provincie Groningen, 9700 RB, Netherlands

  • Hospital Clínico Universitario de Santiago

    RECRUITING

    Santiago, 15706, Spain

  • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

    NOT_YET_RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Istituto G Gaslini Ospedale Pediatrico IRCCS

    RECRUITING

    Genoa, 16147, Italy

  • McGill University Health Center

    RECRUITING

    Montreal, Quebec, H4A3J1, Canada

  • Osaka City General Hospital

    RECRUITING

    Osaka, 534-0021, Japan

  • Primary Children's Hospital

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84108, United States

  • Rigshospitalet

    RECRUITING

    Copenhagen, 2700, Denmark

  • The Cleveland Clinic Foundation

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

  • University of Connecticut Health Center

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Naples

    RECRUITING

    Naples, Campania, 80131, Italy

  • University of Texas Health Science Center at Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • Universitätsklinikum Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE IA