19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

3 trials found

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	NCT03643562 (ClinicalTrials.gov)	June 18, 2018	19/8/2018	Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System	Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)	Niemann-Pick Type C Disease	Drug: Adrabetadex	Mandos LLC	NULL	Active, not recruiting	4 Years	N/A	All	60	Phase 3	United States
2	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
3	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