19. Lysosomal storage disease Clinical trials / Disease details

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

2 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	NCT01853852 (ClinicalTrials.gov)	September 2011	10/11/2011	A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects	A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects	Fabry Disease	Drug: GR181413A/AT1001 solution;Drug: GR181413A/AT1001 capsule;Other: Potable water;Drug: Placebo capsule	Amicus Therapeutics	NULL	Completed	20 Years	55 Years	Both	14	Phase 1	Australia
2	NCT00001972 (ClinicalTrials.gov)	September 1994	18/1/2000	PET Scan of Brain Metabolism in Relation to Age and Disease	Positron Emission Tomography Imaging of Human Brain Phospholipid Metabolism in Relation to Age and Disease	Alzheimer's Disease;Brain Neoplasm;Niemann Pick Disease	Drug: 15 O Water	National Institute of Neurological Disorders and Stroke (NINDS)	NULL	Completed	N/A	N/A	Both	123	N/A	United States