Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Status:
Completed
Trial end date:
2010-11-01
Target enrollment:
Participant gender:
Summary
The objectives of this clinical trial are to assess the safety and tolerability, as well as
efficacy, of a stepwise dosing regimen of pyrimethamine, starting at 25 mg/day, given as a
single dose daily for 4 weeks in patients affected with chronic Tay-Sachs or Sandhoff
variants.