CompletedPhase 1/2

SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)

United States, Germany, Italy32 participantsStarted 2009-11

Plain-language summary

The objective of this study will be to evaluate the safety, tolerability and effect on SOD1 levels by pyrimethamine in patients with familial amyotrophic lateral sclerosis.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. ALS diagnosed as probable, laboratory supported probable or definite according to the World Federation of Neurology El Escorial criteria \[Brooks et al. 2000\]
✓. Age 18 or older
✓. Capable of providing informed consent and complying with trial procedures
✓. SOD1 mutation confirmation by study team
✓. Not taking Riluzole (Rilutek) or on a stable dose for 30 days
✓. Not taking Coenzyme QR10R or on a stable dose and brand for 30 days

Exclusion criteria

✕. History or evidence of malabsorption syndromes
✕. Exposure to any experimental agent within 30 days of onset of this protocol
✕. Women who are pregnant or planning to become pregnant
✕. Women of childbearing potential not practicing contraception
✕. Women who are breastfeeding
✕. Enrollment in another research study within 30 days of or during this trial
✕. Alcoholism
✕. Patients taking phenytoin (Dilantin) or other therapy affecting folate levels

What they're measuring

Mean Change in SOD1 CSF

Timeframe: baseline, Visit 6 week 18, end of study

Trial details

NCT IDNCT01083667

SponsorWeill Medical College of Cornell University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2014-12

Results submitted2017-02-17

View on ClinicalTrials.gov More Familial Amyotrophic Lateral Sclerosis trials