Update (June 16, 2014):
This trial iw now closed to new participants.
Name: Pyrimethamine. This drug is approved by the U.S. Food and Drug Administration for the treatment of malaria and toxoplasmosis; it is marketed under the brand name Daraprim.
How it works: Pyrimethamine reduces levels of SOD1 protein. Although mutations in the SOD1 gene primarily are associated with the familial form of ALS, toxicity from misfolded SOD1 protein has been seen in both familial and sporadic (in which there is no known family history) ALS.
Skeletal formula of pyrimethamine
(courtesy of ChemSpider).
Purpose of trial: To evaluate the safety and tolerability, and effect of pyrimethamine on SOD1 protein levels in people with familial ALS.
Type of trial: Phase 1-2 in people with familial ALS caused by mutations in the SOD1 gene.
- Weill Cornell Medical Center/New York Presbyterian Hospital, New York
- Methodist Neurological Institute, Houston
- Milano Neurological Institute, Milan, Italy
- Umea University, Umea, Sweden
- The primary objective is to determine if people with familial ALS taking pyrimethamine will show a 15 percent or greater decline in SOD1 levels in the cerebrospinal fluid.
- Secondary outcome measures are: safety and tolerability; adverse events; blood concentrations of SOD1 and pyrimethamine; and a decline in SOD1 levels over the course of the study.
- Investigators also will assess the feasibility of proceeding to phase 2-3 trials of pyrimethamine in ALS.
Currently recruiting? This study is currently recruiting participants.
Background info: In a phase 1 pilot study, investigators found that treatment with pyrimethamine was associated with decreased levels of SOD1 protein in people with ALS. See also: Pyrimethamine Decreases Levels of SOD1 in Leukocytes and Cerebrospinal Fluid of ALS Patients: A Phase 1 Pilot Study.
Additional info: For trial information, enrollment criteria and contact information, see SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS).
To read about the other ALS clinical trials:
Note: The effect of these drugs on people with ALS is still unknown. Individuals are strongly advised not to use these drugs to treat ALS until they have been proven safe and effective, and have been approved for use in ALS. Always consult with your physician before adding any medications to your treatment plan.
About Clinical Trials
A clinical trial is a test, in humans, of an experimental treatment. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit, or even harm, may occur.
MDA has no ability to influence who is chosen to participate in a clinical trial.
To learn more, see Learn About Clinical Studies and Being a Co-Adventurer, which is about neuromuscular disease clinical trials. To see a continuously updated database of clinical trials, go to ClinicalTrials.gov.