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19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
6 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

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