Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check

NCT ID NCT03970278

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

First seen Mar 21, 2026 · Last updated May 15, 2026 · Updated 5 times

Summary

This study checked the long-term safety of a one-time gene therapy (DTX401) in 12 adults with glycogen storage disease type Ia, a rare condition that causes dangerously low blood sugar. Participants were followed for side effects and how well their bodies handled fasting without severe drops in blood sugar. The goal was to see if the treatment remains safe over time and helps control the disease.

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Contacts and locations

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Locations

Complejo Hospitalario Universitario de Santiago

Santiago de Compostela, A Coruna, 15706, Spain
Michigan Medicine University of Michigan

Ann Arbor, Michigan, 48109, United States
Montreal Children Hospital, McGill University Health Centre

Montreal, Quebec, H4A3J1, Canada
UCONN Health

Farmington, Connecticut, 06030-3213, United States
University Medical Center Groningen

Groningen, 9700RB, Netherlands
University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE IA GLYCOGEN STORAGE DISEASE TYPE IA VON GIERKE'S DISEASE (GSD TYPE IA)