Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

NCT01102686 · clinicaltrials.gov ↗

PHASE1,PHASE2

Phase

COMPLETED

Status

20

Enrollment

OTHER

Sponsor class

Conditions

Gangliosidoses, GM2
Sandhoff Disease
Tay-Sachs Disease

Interventions

DRUG: Pyrimethamine
DRUG: Leucovorin

Sponsor

The Hospital for Sick Children