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 | JPRN-UMIN000023517 | 2016/10/01 | 01/10/2016 | A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C | A study on the efficacy and safety of cyclodextrin intrathecal long-term administration for Niemann-Pick disease type C - IT-HPBCD therapy for NPC (Long-term) | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 20mg/kg/dose, every 3-4 weeks, for three years | Nara Medical University | NULL | Pending | 4years-old | 6years-old | Male and Female | 1 | Not selected | Japan |
| 2 | JPRN-UMIN000020032 | 2014/12/19 | 02/12/2015 | Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) | Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (NPC) - IT-HPBCD therapy in adult-onset NPC | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 100-400mg, every 4 week, two years | Jichi Medical University, Saitama Medical Center | Saga UniversityKumamoto UniversityTohoku UniversityInstitute for Neurological Disorders | Complete: follow-up continuing | 20years-old | Not applicable | Male and Female | 2 | Not applicable | Japan |
| 3 | JPRN-UMIN000023094 | 2014/08/01 | 10/07/2016 | A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C | A study on the safety and efficacy of cyclodextrin intrathecal administration in Niemann-Pick disease type C - IT-HPBCD therapy for NPC | Niemann-Pick disease type C | 2-hydroxypropyl-beta-cyclodextrin (HPBCD) 5-20mg/kg, every 4 weeks, for two years | Nara Medical University | Saga UniversityKumamoto University | Complete: follow-up continuing | 1years-old | 25years-old | Male and Female | 1 | Not selected | Japan |