Sanofi: Gaucher Disease trials

The registry lists 24 Gaucher Disease studies with Sanofi as lead sponsor. 3 are open to participants now and 6 have results posted.

Phase 1 6Phase 2 2Phase 3 7Phase 4 3No phase 6(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• The Efficacy and Safety of Venglustat Compared With Cerezyme in Pediatric Participants 2 to 11 Years of Age With Gaucher Disease Type 3 (GD3)Not yet recruitingNCT07927140Phase 3
• Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).AvailableNCT07675031
• International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registryRecruitingNCT00358943
• Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 StudiesCompletedNCT02536755Phase 3Results posted
• A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)CompletedNCT01074944Phase 3Results posted
• A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)CompletedNCT00943111Phase 3Results posted
• A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)CompletedNCT00891202Phase 3Results posted
• Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher DiseaseCompletedNCT00364858Phase 4Results posted
• A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher PatientsCompletedNCT00358150Phase 2Results posted
• A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the MouthCompletedNCT06193304Phase 1
• A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor MetabolizersCompletedNCT06188325Phase 1
• A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male ParticipantsCompletedNCT06143904Phase 1
• Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)CompletedNCT05908656Not applicable
• Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3Active, not recruitingNCT05222906Phase 3
• A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged SpleenCompletedNCT04845958
• Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ⅢCompletedNCT04656600Phase 4
• A National Study in Patients With Unexplained SplenomegalyCompletedNCT04430881
• Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3CompletedNCT03485677Phase 3
• Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy ExtensionActive, not recruitingNCT02843035Phase 2
• A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat TartrateCompletedNCT02536937Phase 1
• A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat TartrateCompletedNCT02536911Phase 1
• Taste Evaluation of Different Liquid Formulations With EliglustatCompletedNCT02422654Phase 1
• A Screening Study Evaluating Disease Status of Gaucher Type I PatientsWithdrawnNCT00795197
• A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.CompletedNCT00365131Phase 4