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

Glycogen Storage Disease Type IA Clinical Trial

Official title:

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03970278
• Other study ID #: 401GSDIA02
• Secondary ID: 2018-004473-27
• Status: Active, not recruiting
• Start date: July 15, 2019
• Est. completion date: December 2026

Study information

• Verified date: May 2024
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 12
• Est. completion date: December 2026
• Est. primary completion date: December 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

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.

Related Conditions & MeSH terms

• Disease
• Glycogen Storage Disease
• Glycogen Storage Disease Type I
• Glycogen Storage Disease Type IA
• Metabolic Diseases
• Von Gierke's Disease (GSD Type Ia)

Intervention

Other:
• No intervention
No intervention

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Canada,  Netherlands,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs		Up to 312 weeks following DTX401 administration
Secondary	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