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

> **NCT01102686** · PHASE1,PHASE2 · COMPLETED · sponsor: **The Hospital for Sick Children** · enrollment: 20 (estimated)

## Conditions studied

- Gangliosidoses, GM2
- Sandhoff Disease
- Tay-Sachs Disease

## Interventions

- **DRUG:** Pyrimethamine
- **DRUG:** Leucovorin

## Key facts

- **NCT ID:** NCT01102686
- **Lead sponsor:** The Hospital for Sick Children
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2009-08
- **Primary completion:** 2010-11
- **Final completion:** 2010-11
- **Target enrollment:** 20 (ESTIMATED)
- **Last updated:** 2012-02-23


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01102686

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01102686, "Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT01102686. Licensed CC0.

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