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

Plain-language summary

This study was designed to determine the long-term safety of DTX401 after a single intravenous (IV) dose in adults with glycogen storage disease type Ia (GSDIa). It is a follow-up study in which no new treatment is given. To take part, participants must have previously received DTX401 in study 401GSDIA01, provide written informed consent, and be able to attend all scheduled study visits.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Ultragenyx Pharmaceutical Inc

Intervention

No intervention

Start date

7/15/2019

Estimated completion

2/25/2025

Countries

United States, Canada, Netherlands, Spain

Related genes

Locations

• Farmington, United States
• Ann Arbor, United States
• Houston, United States
• Montreal, Canada
• Groningen, Netherlands
• Santiago de Compostela, Spain

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. Received DTX401 in study 401GSDIA01.
2. 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.
3. Willing and able to comply with all scheduled study visits, procedures, and requirements.

Exclusion Criteria:

1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
2. 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.