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 | NCT03643562 (ClinicalTrials.gov) | June 18, 2018 | 19/8/2018 | Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System | Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC) | Niemann-Pick Type C Disease | Drug: Adrabetadex | Mandos LLC | NULL | Completed | 4 Years | N/A | All | 27 | Phase 3 | United States |