Vts-270    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病6

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.
6 / 784 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03687476
(ClinicalTrials.gov)
May 20206/8/2018Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) DiseaseAn 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) DiseaseNiemann-Pick Disease, Type CDrug: VTS-270Vtesse, Inc., a Mallinckrodt Pharmaceuticals CompanyNULLWithdrawnN/A4 YearsAll0Phase 2NULL
2NCT03887533
(ClinicalTrials.gov)
January 6, 202022/3/2019Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1Niemann-Pick Disease, Type C1Drug: VTS-270Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)NULLRecruiting3 Years60 YearsAll30Phase 1;Phase 2United States
3NCT03879655
(ClinicalTrials.gov)
December 2, 201912/12/2018Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1A 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 VTS301Niemann-Pick Disease, Type CDrug: VTS-270Vtesse, Inc., a Mallinckrodt Pharmaceuticals CompanyNULLActive, not recruiting4 Years21 YearsAll7Phase 2;Phase 3Costa Rica
4NCT03471143
(ClinicalTrials.gov)
February 22, 20197/3/2018Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CPhase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CNiemann-Pick Disease, Type CDrug: 2-Hydroxypropyl-Beta-CyclodextrinWashington University School of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)RecruitingN/A6 MonthsAll12Phase 1;Phase 2United States
5NCT02534844
(ClinicalTrials.gov)
October 201518/8/2015VTS-270 to Treat Niemann-Pick Type C1 (NPC1) DiseaseA 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) DiseaseNiemann-Pick Disease, Type CDrug: VTS-270;Other: ShamVtesse, Inc., a Mallinckrodt Pharmaceuticals CompanyNULLActive, not recruiting4 Years21 YearsAll51Phase 2;Phase 3United States;Australia;France;Germany;New Zealand;Singapore;Spain;Turkey;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01747135
(ClinicalTrials.gov)
January 20138/12/2012Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 DiseaseHydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 DiseaseNiemann-Pick Disease, Type C1Drug: VTS-270Vtesse, Inc., a Mallinckrodt Pharmaceuticals CompanyNULLCompleted2 Years25 YearsAll14Phase 1United States