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

Termination Rate

3.0%

1 terminated out of 33 trials

Success Rate

95.5%

+8.9% vs benchmark

Late-Stage Pipeline

33%

11 trials in Phase 3/4

Results Transparency

57%

12 of 21 completed with results

Key Signals

12 with results95% success

Data Visualizations

Phase Distribution

27Total
Not Applicable (2)
P 1 (5)
P 2 (9)
P 3 (8)
P 4 (3)

Trial Status

Completed21
Recruiting4
Unknown3
Withdrawn2
Active Not Recruiting2
Terminated1

Trial Success Rate

95.5%

Benchmark: 86.5%

Based on 21 completed trials

Clinical Trials (33)

Showing 20 of 20 trials
NCT05253560RecruitingPrimary

Prodromal Parkinsonian Features in GBA1 Mutation Carriers

NCT07223944Phase 3RecruitingPrimary

A Gaucher Disease Gene Therapy Trial With FLT201

NCT05487599Phase 1Recruiting

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT02583672Phase 2CompletedPrimary

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

NCT06818838Phase 1RecruitingPrimary

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

NCT06545136Phase 1Active Not RecruitingPrimary

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

NCT05324943Phase 1CompletedPrimary

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05687474Completed

Baby Detect : Genomic Newborn Screening

NCT02843035Phase 2Active Not RecruitingPrimary

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

NCT00319046Phase 3CompletedPrimary

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

NCT06050967Phase 2CompletedPrimary

A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.

NCT03950050Phase 2CompletedPrimary

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

NCT03746587Phase 2TerminatedPrimary

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

NCT04353466Not ApplicableCompletedPrimary

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

NCT04120506Phase 4CompletedPrimary

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

NCT05447494Phase 1UnknownPrimary

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

NCT00430625Phase 3CompletedPrimary

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

NCT03333447CompletedPrimary

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)

NCT00635427Phase 3CompletedPrimary

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00553631Phase 3CompletedPrimary

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

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