Long-Term Observational Monitoring of Glycogen Storage Disease Type Ia Patients Treated with DTX401 Evaluating Safety and Effectiveness Over 10 Years

What this Trial Is About

This observational study follows patients with Glycogen Storage Disease Type Ia GSDIa who have received DTX401 treatment. The main goal is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after administration. It includes participants from previous clinical studies and those treated in a post-marketing setting. Participants are observed without receiving any new investigational treatment as part of this study. The program tracks two groups those who got DTX401 in prior clinical trials and those who received it after the drug became available commercially. Monitoring covers a range of outcomes related to safety and treatment effects over a decade. During the study, participants undergo regular evaluations including assessments of serious adverse events, pregnancy outcomes, dietary intake, glucose levels, hypoglycemic events, metabolic parameters, hospital visits, and quality of life. Data collection involves medical records review, patient interviews, questionnaires, and laboratory tests. The total participation time spans at least 10 years to provide comprehensive long-term information on DTX401 treatment.

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program