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

Completed

Conditions studied: Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)

In brief

The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

Key facts

Study ID: NCT03970278
Run by: Ultragenyx Pharmaceutical Inc
People needed: 12
Starts: 2019-07-15
Expected to finish: 2025-02-25
Last updated by the study team: 2026-03-18

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Received DTX401 in study 401GSDIA01.
Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
Willing and able to comply with all scheduled study visits, procedures, and requirements.

You may not qualify if…

Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.

Where it is running

UCONN Health — Farmington, Connecticut, United States
Michigan Medicine University of Michigan — Ann Arbor, Michigan, United States
University of Texas Health Science Center at Houston — Houston, Texas, United States
Montreal Children Hospital, McGill University Health Centre — Montreal, Quebec, Canada
University Medical Center Groningen — Groningen, Netherlands
Complejo Hospitalario Universitario de Santiago — Santiago de Compostela, A Coruna, Spain

Full record on ClinicalTrials.gov

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