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

Completed

• Conditions: Von Gierke's Disease (GSD Type Ia); Glycogen Storage Disease Type IA

• Registration Number: NCT03970278
• Lead Sponsor: Ultragenyx Pharmaceutical Inc

Brief 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.

Detailed Description

Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02.

Study Timeline

• Study First Submitted: 2019-05-29
• Study First Submitted QC: 2019-05-29
• Study First Posted: 2019-05-31
• Study Start: 2019-07-15
• Primary Completion: 2025-02-25
• Study Completion: 2025-02-25
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-03
• Last Update Posted: 2025-03-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs	Up to 312 weeks following DTX401 administration

Secondary Outcome Measures

Name	Time	Method
Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 312 weeks	Up to 312 weeks following DTX401 administration

Trial Locations

Locations (6)

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; 🇨🇦 Montréal, Quebec, Canada

University Medical Center Groningen; 🇳🇱 Groningen, Netherlands

Complejo Hospitalario Universitario de Santiago; 🇪🇸 Santiago De Compostela, A Coruna, Spain