SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS) (NCT01083667) | Clinical Trial Compass
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 range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Mean Change in SOD1 CSF
Timeframe: baseline, Visit 6 week 18, end of study
Trial details
NCT IDNCT01083667
SponsorWeill Medical College of Cornell University