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Pyrimethamine for Late-Onset GM2-gangliosidosis
NCT01102686Phase 2
Key facts
- Status
- Completed
- Start
- Aug 2009
- Primary completion
- Nov 2010
- Countries
- 1
- Sponsor
- SickKids
- Modality
- Small Molecules
- Drugs
- pyrimethamine
- Conditions
- Tay-Sachs disease (TSD)Sandhoff disease
01
Overview
Phase 2, open-label, single-arm trial of pyrimethamine in Tay-Sachs disease (TSD) and Sandhoff disease, sponsored by SickKids. Primary endpoint: Efficacy of pyrimethamine at 18 weeks. Started Aug 2009; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
Pyrimethamine
Stepwise dose escalation of Pyrimethamine with Leucovorin
03
Who it enrols
Adult patients (>17 years) with biochemically and genetically confirmed chronic GM2-gangliosidosis (Tay-Sachs or Sandhoff) with HEXA or HEXB mutations responsive to pyrimethamine in vitro.
04
Endpoints
Primary
Efficacy of pyrimethamine
Secondary
Pyrimethamine Blood levels
Pyrimethamine efficacy
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