19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
1 trial found
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1NCT03687476
(ClinicalTrials.gov)
May 20206/8/2018Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) DiseaseAn Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) DiseaseNiemann-Pick Disease, Type CDrug: VTS-270Vtesse, Inc., a Mallinckrodt Pharmaceuticals CompanyNULLWithdrawnN/A4 YearsAll0Phase 2NULL