19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| 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 | NCT02534844 (ClinicalTrials.gov) | October 2015 | 18/8/2015 | VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease | A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease | Niemann-Pick Disease, Type C | Drug: Parts A/B: Adrabetadex;Other: Parts A/B: Sham Control | Mandos LLC | NULL | Active, not recruiting | 4 Years | 21 Years | All | 51 | Phase 2/Phase 3 | United States;Australia;France;Germany;New Zealand;Singapore;Spain;Turkey;United Kingdom |