Indication
Gaucher's Disease
15 clinical trials
18 products
Clinical trial
A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher DiseaseStatus: Active (not recruiting), Estimated PCD: 2024-08-25
Product
Velaglucerase AlfaProduct
FLT201Clinical trial
A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)Status: Recruiting, Estimated PCD: 2024-11-30
Product
AVR-RD-02Clinical trial
The Guard1 Trial, an Open-Label, Multinational Phase 1/2 Study of the Safety and Efficacy of Ex Vivo, Lentiviral Vector-Mediated Gene Therapy AVR-RD-02 for Subjects With Type 1 Gaucher DiseaseStatus: Terminated, Estimated PCD: 2023-08-21
Clinical trial
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)Status: Recruiting, Estimated PCD: 2030-10-02
Product
LY3884961Clinical trial
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher DiseaseStatus: Active (not recruiting), Estimated PCD: 2028-05-01
Product
SirolimusProduct
MethylprednisoloneProduct
PrednisoneClinical trial
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher DiseaseStatus: Completed, Estimated PCD: 2014-05-01
Clinical trial
Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3Status: Terminated, Estimated PCD: 2020-04-01
Product
PlaceboProduct
ArimoclomolProduct
Taliglucerase alfaProduct
Gene therapyClinical trial
Guard3: An Open-label, Parallel-arm, Randomized, Controlled, Phase 2/Phase 3 Study Evaluating the Efficacy and Safety of Autologous HSC Gene Therapy, AVR-RD-02, Compared to ERT for Gaucher Disease Type 3 in Participants Aged 2 to 25Status: Withdrawn, Estimated PCD: 2027-12-01
Product
Enzyme Replacement AgentProduct
15-0 H20Clinical trial
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher DiseaseStatus: Completed, Estimated PCD: 2023-07-30
Product
ElelysoClinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01
Clinical trial
Role of Vitamin E as an Adjuvant Therapy for Patients With Gaucher Disease Under Enzyme Replacement Therapy in Relation to Oxidative StressStatus: Completed, Estimated PCD: 2023-09-05
Product
Vitamin EClinical trial
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs).Status: Recruiting, Estimated PCD: 2031-07-01
Product
AldurazymeClinical trial
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement TherapyStatus: Completed, Estimated PCD: 2022-11-30
Product
AmbroxolClinical trial
A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02Status: Active (not recruiting), Estimated PCD: 2037-06-01