A clinical study to learn about the effects of a virus that transfers the gene for Glucose- 6-Phosphatase (G6Pase) in adults with Glycogen Storage Disease Type Ia
- Conditions
- Glycogen storage disease type IaMedDRA version: 20.1Level: LLTClassification code: 10056911Term: Glycogen storage disease type IA Class: 10010331Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- CTIS2023-504004-29-00
- Lead Sponsor
- ltragenyx Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 12
Individuals eligible to participate in this study must meet all of the following criteria: 1. 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
Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study: 1. 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method