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)
Verified date | May 2024 |
Source | Ultragenyx Pharmaceutical Inc |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
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. |
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 |
Lead Sponsor | Collaborator |
---|---|
Ultragenyx Pharmaceutical Inc |
United States, Canada, Netherlands, Spain,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Active, not recruiting |
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