Not applicableStudy completedNCT04997772What this trial is testingDigital Health Platform Customized for Patients With Gaucher DiseaseWho this might be right forGaucher DiseasePatient Reported Outcome Shoshana Vilk 80
Early research (Phase 1)Study completedNCT00004294What this trial is testingPhase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher DiseaseWho this might be right forGaucher's Disease National Center for Research Resources (NCRR) 15
Large-scale testing (Phase 3)Study completedNCT01614574What this trial is testingStudy of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher DiseaseWho this might be right forGaucher Disease Shire 6
Not applicableEnrolling By InvitationNCT05368038What this trial is testingScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramWho this might be right forAcid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more Albert Einstein College of Medicine 100,000
Large-scale testing (Phase 3)Study completedNCT02536755What this trial is testingPhase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 StudiesWho this might be right forGaucher Disease Genzyme, a Sanofi Company 31
Large-scale testing (Phase 3)Study completedNCT01422187What this trial is testingExtension Study of Taliglucerase Alfa in Adult Subjects With Gaucher DiseaseWho this might be right forGaucher Disease Pfizer 19
Post-approval studies (Phase 4)Study completedNCT04120506What this trial is testingLong Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)Who this might be right forGaucher Disease, Type 1 Shaare Zedek Medical Center 15
Not applicableActive Not RecruitingNCT06488261What this trial is testingLong Term Follow-Up for Safety of AVR-RD-02Who this might be right forGaucher Disease John Bernat 2
Not applicableStudy completedNCT02785744What this trial is testingGenzyme Osteopenia/Osteoporosis StudyWho this might be right forGaucher Disease NYU Langone Health 76
Testing effectiveness (Phase 2)Study completedNCT00813865What this trial is testingA Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsWho this might be right forGaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1 Amicus Therapeutics 8
Post-approval studies (Phase 4)Study completedNCT04002830What this trial is testingA Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher DiseaseWho this might be right forGaucher Disease, Type 3 Ari Zimran 14
Not applicableLooking for participantsNCT05619900What this trial is testingRegistry of Patients Diagnosed With Lysosomal Storage DiseasesWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more University of California, San Francisco 250
Not applicableUnknownNCT02120235What this trial is testingInvestigating Lysosomal Storage Diseases in Minority GroupsWho this might be right forLysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more O & O Alpan LLC 20,000
Post-approval studies (Phase 4)Study completedNCT04656600What this trial is testingStudy to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ⅢWho this might be right forGaucher's Disease Sanofi 12
Testing effectiveness (Phase 2)Looking for participantsNCT06818838What this trial is testingA Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher DiseaseWho this might be right forGaucher Disease Type 1 Lingyi Biotech Co., Ltd. 12
Testing effectiveness (Phase 2)Active Not RecruitingNCT04127578What this trial is testingPhase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)Who this might be right forParkinson Disease Prevail Therapeutics 32
Not applicableStudy completedNCT01439607What this trial is testingCellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher DiseaseWho this might be right forGaucher Disease Assistance Publique - Hôpitaux de Paris 30
Not applicableLooking for participantsNCT06573723What this trial is testingInstitutional Registry of Rare DiseasesWho this might be right forRare DiseasesAmyloidosisSarcoidosis+23 more Hospital Italiano de Buenos Aires 380
Testing effectiveness (Phase 2)Active Not RecruitingNCT04411654What this trial is testingPhase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)Who this might be right forGaucher Disease, Type 2 Prevail Therapeutics 7
Very early researchLooking for participantsNCT06272149What this trial is testingAn Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher DiseaseWho this might be right forType II Gaucher Disease Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 6