Fabry Disease
212
34
46
105
Key Insights
Highlights
Success Rate
89% trial completion (above average)
Published Results
46 trials with published results (22%)
Research Maturity
105 completed trials (50% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.1%
13 terminated out of 212 trials
89.0%
+2.5% vs benchmark
18%
39 trials in Phase 3/4
44%
46 of 105 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 105 completed trials
Clinical Trials (212)
Quality of Life in Untreated Fabry
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Fabry Disease Registry & Pregnancy Sub-registry
A Study of Patients With Fabry Disease (US Specific)
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
Natural History in Fabry Disease With IVS4+919G>A Mutations
Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
T1 Mapping in Fabry Disease
Care Pathway for Patients With Fabry's Disease (Fabry-PATH)
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Early Detection of Fabry Disease
A Real-world Wearables Study in Fabry Disease.
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting