Gaucher Disease
89
12
15
50
Key Insights
Highlights
Success Rate
94% trial completion (above average)
Published Results
21 trials with published results (24%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
3.4%
3 terminated out of 89 trials
94.3%
+7.8% vs benchmark
18%
16 trials in Phase 3/4
42%
21 of 50 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 50 completed trials
Clinical Trials (89)
Genetic Studies of Lysosomal Storage Disorders
Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Gaucher Disease Outcome Survey (GOS)
World Data on Ambroxol for Patients With GD and GBA Related PD
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Longitudinal Study of Neurodegenerative Disorders
A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease
Institutional Registry of Rare Diseases
A Long-term Follow-up Study of Gaucher Disease
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
Long Term Follow-Up for Safety of AVR-RD-02
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Omics Gaucher Study: Multiomic Approach
The GBA Multimodal Study in Parkinson's Disease
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion