Gaucher's Disease
14
0
0
12
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 14 trials
100.0%
+13.5% vs benchmark
7%
1 trials in Phase 3/4
17%
2 of 12 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 12 completed trials
Clinical Trials (14)
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers
A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth
Human Placental-Derived Stem Cell Transplantation
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
Stem Cell Transplant for Inborn Errors of Metabolism
Effects of Enzyme Replacement in Gaucher's Disease
Bone Response to Enzyme Replacement in Gaucher's Disease
Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease