CompletedNot Applicable

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

United States, Canada, Netherlands12 participantsStarted 2019-07-15

Plain-language summary

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.

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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.

Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs

Timeframe: From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329

NCT IDNCT03970278

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-02-25

Results submitted2026-02-24