Gene therapy for rare metabolic 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 23, 2026 · Updated 8 times

Summary

This study followed 12 adults with glycogen storage disease type Ia (GSDIa) who had received a single dose of gene therapy (DTX401) in an earlier trial. The main goal was to see if the treatment was safe over the long term. Researchers also checked how well it helped control blood sugar levels during fasting.

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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)