Fabry Disease
215
35
47
106
Key Insights
Highlights
Success Rate
88% trial completion (above average)
Published Results
48 trials with published results (22%)
Research Maturity
106 completed trials (49% of total)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
7.0%
15 terminated out of 215 trials
87.6%
+1.0% vs benchmark
19%
40 trials in Phase 3/4
45%
48 of 106 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 106 completed trials
Clinical Trials (215)
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Anderson-Fabry Disease Fitness Improvement Training
A Study of Patients With Fabry Disease (US Specific)
A Study of Replagal in Children and Adults With Fabry Disease in India
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
A Fabry Disease Gene Therapy Study
RAre, But Not aLone: a Large Italian Network to Empower the Impervious diaGNostic Pathway of Rare cerEbrovascular Diseases (ALIGNED)
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Long-Term Follow-up of Subjects Who Were Treated With ST-920
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
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
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease