19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details

臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191

2 trials found

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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	NCT01102686 (ClinicalTrials.gov)	August 2009	16/10/2009	Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)	Proposed Investigator-Initiated Clinical Trial of Pyrimethamineas a Treatment for Late-Onset GM2-gan ...	Gangliosidoses, GM2;Sandhoff Disease;Tay-Sachs Disease	Drug: Pyrimethamine;Drug: Leucovorin	The Hospital for Sick Children	NULL	Completed	17 Years	N/A	Both	20	Phase 1/Phase 2	Canada
2	NCT00679744 (ClinicalTrials.gov)	May 2008	15/5/2008	A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis	A Dose-Escalated, Double-Blind, Placebo-Controlled, Randomized Phase I Clinical Trial of Pyrimethami ...	G(M2) Ganglioside;Tay-Sachs Disease Ganglioside;Sandhoff Disease Ganglioside	Drug: Pyrimethamine	Exsar Corporation	University Hospital Case Medical Center;New York University School of Medicine;The Hospital for Sick ...	Withdrawn	18 Years	N/A	Both	0	Phase 1	United States;Canada

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