| Not Yet Recruiting | A Gaucher Disease Gene Therapy Trial With FLT201 NCT07223944 | Spur Therapeutics | Phase 3 |
| Recruiting | A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease NCT06818838 | Lingyi Biotech Co., Ltd. | Phase 1 / Phase 2 |
| Active Not Recruiting | Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) NCT06545136 | Spur Therapeutics | Phase 1 / Phase 2 |
| Recruiting | A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) NCT05487599 | Prevail Therapeutics | Phase 1 / Phase 2 |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Unknown | Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease NCT05447494 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Phase 1 / Phase 2 |
| Completed | A Gene Therapy Study in Patients With Gaucher Disease Type 1 NCT05324943 | Spur Therapeutics | Phase 1 |
| Completed | A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacem NCT06050967 | Hadassah Medical Organization | Phase 2 |
| Unknown | Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease NCT04055831 | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | — |
| Completed | Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therap NCT03950050 | Shaare Zedek Medical Center | Phase 2 |
| Terminated | Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 NCT03746587 | ZevraDenmark | Phase 2 |
| Unknown | Prodromal Parkinsonian Features in GBA1 Mutation Carriers NCT05253560 | Shaare Zedek Medical Center | — |
| Completed | Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With NCT03333447 | CEN Biotech | — |
| Active Not Recruiting | Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monother NCT02843035 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs NCT04353466 | Shaare Zedek Medical Center | N/A |
| Completed | Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) NCT04120506 | Shaare Zedek Medical Center | Phase 4 |
| Completed | Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) NCT02583672 | University of Minnesota | Phase 2 |
| Withdrawn | The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease NCT02528617 | Baylor Research Institute | Phase 4 |
| Completed | Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gauche NCT02067247 | Shaare Zedek Medical Center | N/A |
| Completed | Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease NCT02770625 | ISU Abxis Co., Ltd. | Phase 3 |
| Completed | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) NCT00891202 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goa NCT00943111 | Genzyme, a Sanofi Company | Phase 3 |
| Completed | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients NCT00813865 | Amicus Therapeutics | Phase 2 |
| Withdrawn | A Screening Study Evaluating Disease Status of Gaucher Type I Patients NCT00795197 | Genzyme, a Sanofi Company | — |
| Completed | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease NCT00446550 | Amicus Therapeutics | Phase 2 |
| Completed | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease NCT00635427 | Shire | Phase 3 |
| Completed | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Di NCT00553631 | Shire | Phase 3 |
| Completed | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzym NCT00433147 | Amicus Therapeutics | Phase 2 |
| Completed | A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease NCT00430625 | Shire | Phase 3 |
| Completed | A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients NCT00358150 | Genzyme, a Sanofi Company | Phase 2 |
| Completed | Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stabl NCT00319046 | Actelion | Phase 3 |
| Completed | Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease NCT00364858 | Genzyme, a Sanofi Company | Phase 4 |
| Approved For Marketing | Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease NCT00954460 | Shire | — |