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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

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

Inclusion criteria

. 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.

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Trial details

NCT IDNCT03970278

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-02-25

Results submitted2026-02-24

View on ClinicalTrials.gov More Glycogen Storage Disease Type IA trials