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-ganglio ... show all
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Disease | Niemann-Pick Disease, Type C | Drug: VTS-270 | Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company | NULL | Withdrawn | N/A | 4 Years | All | 0 | Phase 2 | NULL |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 | 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 |
| 3 | NCT03893071 (ClinicalTrials.gov) | March 2019 | 20/2/2019 | Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPßCD) in Niemann-Pick Disease Type C | An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP-ß-CD) in Patients With Niemann-Pick Disease Type C (NPC-1) | Niemann-Pick Disease, Type C1 | Drug: Hydroxypropyl-ß-cyclodextrin | CTD Holdings, Inc. | NULL | Recruiting | 18 Years | N/A | All | 12 | Phase 1 | United States |
| 4 | EUCTR2015-005761-23-SE (EUCTR) | 19/01/2017 | 09/09/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Cyclo Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | Italy;United Kingdom;Sweden | |||
| 5 | EUCTR2015-005761-23-GB (EUCTR) | 26/09/2016 | 03/08/2016 | A Phase I/II study to evaluate Trappsol Cyclo (hydroxypropyl-ß-cyclodextrin) in patients with Niemann-Pick disease type C (NPC-1) to assess what the drug does to the body, and what the body does to the drug, and the side effects and benefits experienced by patients | A Phase I/II study to evaluate the safety and pharmacokinetics of intravenous Trappsol Cyclo (HP-ß-CD) in patients with Niemann-Pick disease type C (NPC-1) and the pharmacodynamic effects of treatment upon markers of cholesterol metabolism and clinical outcomes | Niemann-Pick disease type C MedDRA version: 20.0;Level: PT;Classification code 10029403;Term: Niemann-Pick disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | CTD Holdings, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 1;Phase 2 | Italy;United Kingdom;Sweden | |||
| 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 | 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 |