Glycogen Storage Disease Type Ia
11
4
4
6
Key Insights
Highlights
Success Rate
86% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
9.1%
1 terminated out of 11 trials
85.7%
-0.8% vs benchmark
9%
1 trials in Phase 3/4
33%
2 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (11)
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease
Accuracy of Lactate Meter in GSDIa
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia
Study of the Relationship Between Glycogen Storage Disease Type Ia and Inflammatory Bowel Disease
Sleep and Quality of Life in Patients With Glycogen Storage Disease on Standard Versus Modified Uncooked Cornstarch