Gaucher Disease: all 174 studies

Page 2 of 2. Open to participants first (40), then studies with results posted, then the rest; newest registration first within each.

• Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ⅢCompletedNCT04656600Phase 4
• Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2CompletedNCT04470713
• A National Study in Patients With Unexplained SplenomegalyCompletedNCT04430881
• Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in IndiaCompletedNCT04429984
• Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)Active, not recruitingNCT04411654Phase 1/2
• Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTsCompletedNCT04353466Not applicable
• Complement Activation in the Lysosomal Storage DisordersWithdrawnNCT04189601
• Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)Active, not recruitingNCT04127578Phase 1/2
• Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)CompletedNCT04120506Phase 4
• The GBA Multimodal Study in Parkinson's DiseaseCompletedNCT04101968
• A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction TherapiesTerminatedNCT04094181
• Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher DiseaseStatus unknownNCT04055831
• Therapeutic Exercise to Treat Neuropathic PainCompletedNCT04050137Not applicable
• Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement TherapyCompletedNCT03950050Phase 2
• Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave ElastographyTerminatedNCT03896607
• Biomarker Analysis for GBA Associated Parkinson's DiseaseStatus unknownNCT03811496
• Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3TerminatedNCT03746587Phase 2
• Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.Status unknownNCT03721627Phase 4
• Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher DiseaseCompletedNCT03702361Phase 4
• Clinical Audit of Managment of Gausher Disease in ChildrenStatus unknownNCT03590548
• Eliglustat on Gaucher Disease Type IIIBCompletedNCT03519646Not applicable
• Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3CompletedNCT03485677Phase 3
• 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)CompletedNCT03333447
• Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher DiseaseWithdrawnNCT03021941Phase 4
• Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy ExtensionActive, not recruitingNCT02843035Phase 2
• Genzyme Osteopenia/Osteoporosis StudyCompletedNCT02785744
• SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher DiseaseStatus unknownNCT02605603
• Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)CompletedNCT02583672Phase 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
• The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher DiseaseWithdrawnNCT02528617Phase 4
• Miglustat on Gaucher Disease Type IIIBStatus unknownNCT02520934Not applicable
• Taste Evaluation of Different Liquid Formulations With EliglustatCompletedNCT02422654Phase 1
• Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher DiseaseCompletedNCT02416661
• Investigating Lysosomal Storage Diseases in Minority GroupsStatus unknownNCT02120235
• An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112CompletedNCT02107846Phase 2
• Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher DiseaseCompletedNCT02067247Not applicable
• A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher DiseaseCompletedNCT02053896
• Intra-monocyte Imiglucerase Kinetics in Gaucher DiseaseStatus unknownNCT01951989Phase 2
• Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using FibroscanStatus unknownNCT01898325Not applicable
• A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy VolunteersCompletedNCT01881633Phase 1
• Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease PatientsCompletedNCT01747980Phase 1
• Identification of Undiagnosed Gaucher DiseaseStatus unknownNCT01716741Not applicable
• Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 1
• Clinical Trial of Ambroxol in Patients With Type I Gaucher DiseaseSuspendedNCT01463215Phase 1/2
• Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher DiseaseCompletedNCT01439607Not applicable
• Clinical and Pathophysiological Investigations Into Erdheim Chester DiseaseCompletedNCT01417520
• Detecting Dopaminergic Deficits in Individuals At-risk for ParkinsonismCompletedNCT01358474
• Exploration of Immunity in Gaucher DiseaseCompletedNCT01358188
• Home Therapy With VPRIV in Gaucher's DiseaseCompletedNCT01356537
• Thrombocytopathy in Gaucher Disease PatientsStatus unknownNCT01344096
• Biomarker for Gaucher Disease: BioGaucher (BioGaucher)WithdrawnNCT01331642
• Examining the Immune Response in Patients With Gaucher Disease and Hepatitis CStatus unknownNCT01274208
• The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher DiseaseWithdrawnNCT01161914Phase 3
• Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)CompletedNCT01136304
• Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher DiseaseNo longer availableNCT00962260
• Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher DiseaseApproved for marketingNCT00954460
• A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101TerminatedNCT00875160Phase 1
• A Screening Study Evaluating Disease Status of Gaucher Type I PatientsWithdrawnNCT00795197
• A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher DiseaseCompletedNCT00465062
• A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.CompletedNCT00365131Phase 4
• Study to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseCompletedNCT00351156
• Positron Emission Tomography (PET) Imaging in People With Gaucher MutationsCompletedNCT00302146
• Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)CompletedNCT00258778Phase 1
• Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher DiseaseCompletedNCT00244582
• OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher DiseaseCompletedNCT00041535Phase 2
• Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT00005900
• Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's DiseaseCompletedNCT00004488Phase 2
• Phase 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 DiseaseCompletedNCT00004294Phase 1
• Phase II Study of Glucocerebrosidase in Patients With Gaucher DiseaseStatus unknownNCT00004293Phase 2
• Bone Response to Enzyme Replacement in Gaucher's DiseaseCompletedNCT00001416Phase 2
• PEG-Glucocerebrosidase for the Treatment of Gaucher DiseaseCompletedNCT00001410Phase 1
• Effects of Enzyme Replacement in Gaucher's DiseaseCompletedNCT00001289
• Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming CellsCompletedNCT00001234Phase 1

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