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 | NCT03471143 (ClinicalTrials.gov) | February 22, 2019 | 7/3/2018 | Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C | Niemann-Pick Disease, Type C | Drug: 2-Hydroxypropyl-Beta-Cyclodextrin | Washington University School of Medicine | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Active, not recruiting | N/A | 6 Months | All | 3 | Phase 1/Phase 2 | United States |
| 2 | 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 | 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 | |
| 3 | JPRN-UMIN000020032 | 2014/12/19 | 02/12/2015 | Intrathecal 2-hydroxypropyl-beta-cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick disease type C (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 | |
| 4 | 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 | 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 |