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One-Shot gene therapy aims to free patients from constant cornstarch dependence

NCT ID NCT05139316

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

Summary

This study tested a one-time gene therapy injection called DTX401 for people with Glycogen Storage Disease Type Ia (GSDIa), a rare genetic disorder. The main goal was to see if the therapy could safely reduce or eliminate the need for patients to constantly eat cornstarch to keep their blood sugar stable. The trial involved 49 participants who were randomly assigned to receive either the gene therapy or a placebo and were followed for nearly three years.

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Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Duke University

    Durham, North Carolina, 27710, United States

  • Fujita Health University Hospital

    Toyoake, Japan

  • Groningen University

    Groningen, 9700 RB, Netherlands

  • Hospital Clinico Universitario de Santiago

    Santiago de Compostela, A Coruna, 15706, Spain

  • Hospital de Clinicas de Porto Alegre

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

  • Istituto Giannina Gaslini

    Genova, Linguria, 16147, Italy

  • Kumamoto University Hospital

    Kumamoto, Japan

  • McGill University

    Montreal, Quebec, H3H 1P3, Canada

  • Mount Sinai

    The Bronx, New York, 10467, United States

  • Osaka City General Hospital

    Osaka, Japan

  • Primary Children's Hospital

    Salt Lake City, Utah, 84132, United States

  • Righospitalet

    Copenhagen, Capital, 2100, Denmark

  • University Medical Center Eppendorf

    Hamburg, 20251, Germany

  • University of Connecticut Health Center

    Farmington, Connecticut, 06030, United States

  • University of Naples

    Naples, 80131, Italy

  • University of Texas

    Houston, Texas, 77030, United States

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE IA