Key Insights

Highlights

Success Rate

94% trial completion (above average)

Published Results

21 trials with published results (23%)

Research Maturity

51 completed trials (55% of total)

Clinical Risk Assessment

Based on trial outcomes

Low Risk

Score: 27/100

Termination Rate

3.3%

3 terminated out of 92 trials

Success Rate

94.4%

+7.9% vs benchmark

Late-Stage Pipeline

18%

17 trials in Phase 3/4

Results Transparency

41%

21 of 51 completed with results

Key Signals

21 with results94% success

Data Visualizations

Phase Distribution

42Total
Not Applicable (6)
Early P 1 (1)
P 1 (10)
P 2 (8)
P 3 (13)
P 4 (4)

Trial Status

Completed51
Unknown14
Recruiting11
Not Yet Recruiting5
Active Not Recruiting4
Terminated3

Trial Success Rate

94.4%

Benchmark: 86.6%

Based on 51 completed trials

Clinical Trials (92)

Showing 20 of 20 trials
NCT05669729Not Yet RecruitingPrimary

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

NCT07847411Phase 3Not Yet RecruitingPrimary

Study of Velaglucerase Beta ERT in Children With Gaucher Disease

NCT07845188Not Yet RecruitingPrimary

Nutritional Status of Gaucher Disease Patients

NCT00302146Completed

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

NCT00001215Enrolling By Invitation

Genetic Studies of Lysosomal Storage Disorders

NCT03291223RecruitingPrimary

Gaucher Disease Outcome Survey (GOS)

NCT03190837RecruitingPrimary

A Long-term Follow-up Study of Gaucher Disease

NCT04101968Completed

The GBA Multimodal Study in Parkinson's Disease

NCT05487599Phase 1Active Not RecruitingPrimary

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

NCT07758816RecruitingPrimary

Advancing Integrated Therapies for Gaucher Disease

NCT06528080Early Phase 1Active Not RecruitingPrimary

A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease

NCT05368038Enrolling By Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT04388969RecruitingPrimary

World Data on Ambroxol for Patients With GD and GBA Related PD

NCT00358943RecruitingPrimary

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

NCT03625882CompletedPrimary

Survey Study for Velaglucerase Alfa (VPRIV) in Japan

NCT05641103CompletedPrimary

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

NCT05992532RecruitingPrimary

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

NCT03333200Recruiting

Longitudinal Study of Neurodegenerative Disorders

NCT06573723Recruiting

Institutional Registry of Rare Diseases

NCT05843552RecruitingPrimary

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

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