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


臨床試験数 : 854 薬物数 : 727 - (DrugBank : 105) / 標的遺伝子数 : 69 - 標的パスウェイ数 : 190

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01102686
(ClinicalTrials.gov)
August 200916/10/2009Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)Proposed Investigator-Initiated Clinical Trial of Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)Gangliosidoses, GM2;Sandhoff Disease;Tay-Sachs DiseaseDrug: Pyrimethamine;Drug: LeucovorinThe Hospital for Sick ChildrenNULLCompleted17 YearsN/ABoth20Phase 1/Phase 2Canada
2NCT00679744
(ClinicalTrials.gov)
May 200815/5/2008A Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisA Dose-Escalated, Double-Blind, Placebo-Controlled, Randomized Phase I Clinical Trial of Pyrimethamine in Patients Affected With Chronic GM2 Gangliosidosis (Tay-Sachs or Sandhoff Variants)G(M2) Ganglioside;Tay-Sachs Disease Ganglioside;Sandhoff Disease GangliosideDrug: PyrimethamineExsar CorporationUniversity Hospital Case Medical Center;New York University School of Medicine;The Hospital for Sick ChildrenWithdrawn18 YearsN/ABoth0Phase 1United States;Canada