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

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01083667
• Date of registration: 17/12/2009
• Prospective Registration: No
• Primary sponsor: Weill Medical College of Cornell University
• Public title: SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: Phase I/II Study of SOD1 Inhibition by Pyrimethamine in Familial ALS
• Date of first enrolment: November 2009
• Target sample size: 32
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01083667
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Germany, Italy, Sweden, United States

Contacts

• Name: Dale J. Lange, M.D.
• Affiliation: Hospital for Special Surgery/Weill Cornell Medical Center

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Familial Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Pyrimethamine

Primary Outcome(s)

Mean Change in SOD1 CSF [Time Frame: baseline, Visit 6 week 18, end of study]

Secondary Outcome(s)

Appel ALS Score [Time Frame: Week 0, 6, 18, and end of study]

Secondary ID(s)

0903010259

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Muscular Dystrophy Association

Results

• Results available: Yes
• Date Posted: 19/06/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01083667