19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02574286 (ClinicalTrials.gov) | June 29, 2016 | 9/10/2015 | Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease | An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease | Gaucher Disease | Drug: Velaglucerase alfa;Dietary Supplement: Vitamin D | Shire | NULL | Completed | 18 Years | 70 Years | All | 21 | Phase 4 | United States;Israel;Spain;United Kingdom |
| 2 | NCT00635427 (ClinicalTrials.gov) | May 2008 | 6/3/2008 | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease | An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease | Gaucher Disease, Type 1 | Biological: VPRIV® | Shire | NULL | Completed | 2 Years | N/A | All | 95 | Phase 3 | United States;Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom |