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Gene therapy could free GSD ia patients from constant cornstarch

NCT ID NCT05139316

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This Phase 3 trial tested a gene therapy called DTX401 in 49 people with glycogen storage disease type Ia (GSD Ia). The goal was to see if a single infusion could reduce or eliminate the need for frequent cornstarch doses to keep blood sugar stable. Participants were randomly assigned to receive either the gene therapy or a placebo, and their cornstarch intake was measured over 48 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
DTX401 (pariglasgene brecaparvovec) gene therapy
What this could lead to
If successful, this gene therapy could reduce or eliminate the need for frequent cornstarch doses to maintain normal blood sugar levels in people with GSD Ia.
What could go wrong
This is an early-stage Phase 3 trial with only 49 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues, and long-term effects are unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

glucose metabolism disease glycogen storage disease due to glucose-6-phosphatase deficiency type IA glycogen storage disease I

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GLYCOGEN STORAGE DISEASE TYPE IA

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

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