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 | NCT03687476 (ClinicalTrials.gov) | May 2020 | 6/8/2018 | Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease | An 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) Disease | Niemann-Pick Disease, Type C | Drug: VTS-270 | Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company | NULL | Withdrawn | N/A | 4 Years | All | 0 | Phase 2 | NULL |
| 2 | NCT03887533 (ClinicalTrials.gov) | January 6, 2020 | 22/3/2019 | Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 | Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1 | Niemann-Pick Disease, Type C1 | Drug: VTS-270 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | NULL | Completed | 3 Years | 60 Years | All | 2 | Phase 1/Phase 2 | United States |
| 3 | NCT03879655 (ClinicalTrials.gov) | December 2, 2019 | 12/12/2018 | Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 | A Phase 2b/3 Open-label Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 Disease Previously Treated Under Protocol VTS301 | Niemann-Pick Disease, Type C | Drug: VTS-270 | Mandos LLC | NULL | Active, not recruiting | 4 Years | 21 Years | All | 7 | Phase 2/Phase 3 | Costa Rica |
| 4 | 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 |
| 5 | NCT04958642 (ClinicalTrials.gov) | December 23, 2015 | 29/6/2021 | Adrabetadex 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: Adrabetadex | Mandos LLC | NULL | Active, not recruiting | N/A | N/A | All | 66 | Phase 2/Phase 3 | United States;Australia;France;Germany;New Zealand;Singapore;Switzerland;Turkey;United Kingdom |
| 6 | 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 |
| 7 | NCT01747135 (ClinicalTrials.gov) | January 2013 | 8/12/2012 | Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease | Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease | Niemann-Pick Disease, Type C1 | Drug: VTS-270 | Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company | NULL | Completed | 2 Years | 25 Years | All | 14 | Phase 1 | United States |