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

• Status: Completed
• Trial end date: 2016-05-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Weill Medical College of Cornell University
• Collaborator: Muscular Dystrophy Association
• Treatments: Pyrimethamine

Criteria

Inclusion Criteria:

• Subjects with definite, probable, or laboratory supported probable ALS will be eligible.

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. SOD1 mutation confirmation by study team

5. Not taking Riluzole (Rilutek) or on a stable dose for 30 days

6. Not taking Coenzyme QR10R or on a stable dose and brand for 30 days

7. Absence of exclusion criteria

Exclusion Criteria:

1. History or evidence of malabsorption syndromes

2. Exposure to any experimental agent within 30 days of onset of 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 of or during this trial

7. Alcoholism

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); tracheostomy; 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 of or during this trial

19. Incapable of providing informed consent and complying with trial procedures