drugset / Trial / NCT01102686
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Non-randomizedSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2009-08
- Primary completion
- 2010-11
- Completion
- 2010-11
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | pyrimethamine | Small molecule | 100 mg | Oral |
| Background | Leucovorin | Small molecule | 5 mg | — |