Gaucher Disease, Type 1
33
4
6
21
Key Insights
Highlights
Success Rate
95% trial completion (above average)
Published Results
12 trials with published results (36%)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 17/100
3.0%
1 terminated out of 33 trials
95.5%
+8.9% vs benchmark
33%
11 trials in Phase 3/4
57%
12 of 21 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 21 completed trials
Clinical Trials (33)
Prodromal Parkinsonian Features in GBA1 Mutation Carriers
A Gaucher Disease Gene Therapy Trial With FLT201
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)
A Gene Therapy Study in Patients With Gaucher Disease Type 1
Baby Detect : Genomic Newborn Screening
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease