Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

CompletedOBSERVATIONAL

Enrollment

Participants

Timeline

Start Date

July 15, 2019

Primary Completion Date

February 25, 2025

Study Completion Date

February 25, 2025

Conditions

Glycogen Storage Disease Type IAVon Gierke's Disease (GSD Type Ia)

Interventions

OTHER

No intervention

Trial Locations (6)

Complejo Hospitalario Universitario de Santiago, Santiago de Compostela

Michigan Medicine University of Michigan, Ann Arbor

University of Texas Health Science Center at Houston, Houston

UCONN Health, Farmington

Montreal Children Hospital, McGill University Health Centre, Montreal

University Medical Center Groningen, Groningen

All Listed Sponsors

lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT03970278 - Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia | Biotech Hunter | Biotech Hunter