19. ライソゾーム病
［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | Recruiting | 3 Years | 60 Years | All | 30 | 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 | Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company | 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) | Recruiting | N/A | 6 Months | All | 12 | Phase 1;Phase 2 | United States |
| 5 | 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: VTS-270;Other: Sham | Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company | 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 |
| 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 |
| 6 | 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, Inc., a Mallinckrodt Pharmaceuticals Company | NULL | Completed | 2 Years | 25 Years | All | 14 | Phase 1 | United States |