Fabry Disease
213
35
46
106
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Published Results
46 trials with published results (22%)
Research Maturity
106 completed trials (50% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
6.6%
14 terminated out of 213 trials
88.3%
+1.8% vs benchmark
18%
39 trials in Phase 3/4
43%
46 of 106 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 106 completed trials
Clinical Trials (213)
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
A Study of Patients With Fabry Disease (US Specific)
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Fabry Disease Registry & Pregnancy Sub-registry
Study of the Spermatic Characteristics of Patients With Fabry Disease
T1 Mapping in Fabry Disease
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
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