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

NCT01102686 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2012-02-23

No results posted yet for this study

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.

Conditions

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

Interventions

DRUG

Pyrimethamine

Pyrimethamine will be taken orally as a single daily dose of 25 mg/day for 4 weeks, then increasing by 25 mg per dose in three four-week steps, to a final dose of 100 mg/day

DRUG

Leucovorin

To eliminate or minimize potential hematologic effects of Pyrimethamine, Leucovorin is to be co-administered with Pyrimethamine at a dose level of 5 mg per day, given when Pyrimethamine is administered.

Sponsors & Collaborators

  • The Hospital for Sick Children

    lead OTHER

Principal Investigators

  • Joe T Clarke, MD · The Hospital for Sick Children

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-08-31
Primary Completion
2010-11-30
Completion
2010-11-30

Countries

  • Canada

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01102686 on ClinicalTrials.gov