19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) | 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 Pyrimethamine in Patients Affected With Chronic GM2 Gangliosidosis (Tay-Sachs or Sandhoff Variants) | 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| University Hospital Case Medical Center;New York University School of Medicine;The Hospital for Sick Children | Withdrawn | 18 Years | N/A | Both | 0 | Phase 1 | United States;Canada |