Institutional Review Board, Hospital for Special Surgery
July 07, 2010
The safety of study participants is our top priority. The trial is approved and periodically reviewed by an Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.
Before enrolling in a clinical trial, the investigator will explain the purpose of the trial, its expected benefits, any possible risks or side effects, and what your role will be. This is the time to ask questions! If you want to join the trial, you must sign the informed consent documents. You can leave a clinical trial at any time without penalty.
For further information, see Understanding Clinical Trials.
20 patients with mild to moderate Sporadic ALS will receive up to 75 mg of pyrimethamine for a total of 36 weeks. Following Screening and a 12-week titration period, patients will remain on up to 75 mg daily for 24 weeks. This is an open label, single arm dose escalation study.
1. ALS diagnosed as probable, laboratory supported probable or definite according to the World Federation of Neurology El Escorial criteria [Brooks et al. 2000]
2. Age 18 or older
3. Capable of providing informed consent and complying with trial procedures
4. Not taking Riluzole (Rilutek) or on a stable dose for at least 30 days prior to screening
5. Not taking Coenzyme QR10R or on a stable dose and brand for at least 30 days prior to screening
6. Absence of exclusion criteria
1. History or evidence of malabsorption syndromes
2. Exposure to any experimental agent within 30 days prior to screening for this protocol
3. Women who are pregnant or planning to become pregnant
4. Women of childbearing potential not practicing contraception
5. Women who are breastfeeding
6. Enrollment in another research study within 30 days prior to screening for this protocol or during this trial
8. Patients taking phenytoin (Dilantin) or other therapy affecting folate levels
9. Dementia (MMSE <22)
10. Seizure disorder
11. Folate deficiency
12. Megaloblastic anemia
13. Cardiovascular disorder/arrhythmia
14. Impaired kidney function, defined as creatinine levels of 2.5 x ULN
15. Impaired liver function, defined as AST or ALT of 3 X ULN
16. Advanced ALS patients, defined as those with any of the following: forced vital capacity <60% (use of BIPAP is allowed); traecheostomy; or mechanical ventilation
17. Use of any of the following medications: cytosine, arabinoside, methotrexate, daunorubicin, sulfonamides, zidovudine, lorazepam, coumadin, sulfamethoxazole, and trimethoprim
18. Patients taking Lithium within 30 days prior to screening for this protocol or during this trial
19. Incapable of providing informed consent and complying with trial procedures
Nicole Kassebaum, MS
Clinical Research Coordinator
Hospital for Special Surgery