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

Completed · Phase 1/Phase 2

Conditions studied: Familial Amyotrophic Lateral Sclerosis

In brief

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.

Key facts

Study ID

NCT01083667

Run by

Weill Medical College of Cornell University

People needed

32

Starts

2009-11-01

Expected to finish

2016-05-01

Last updated by the study team

2017-06-19

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects with definite, probable, or laboratory supported probable ALS will be eligible.
• 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
• Absence of exclusion criteria

You may not qualify if…

• 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
• Dementia (MMSE <22)
• Seizure disorder
• Folate deficiency
• Megaloblastic anemia
• Cardiovascular disorder/arrhythmia
• Impaired kidney function, defined as creatinine levels of 2.5 x ULN
• Impaired liver function, defined as AST or ALT of 3 X ULN
• Advanced ALS patients, defined as those with any of the following: forced vital capacity <60% (use of BIPAP is allowed); tracheostomy; or mechanical ventilation
• Use of any of the following medications: cytosine, arabinoside, methotrexate, daunorubicin, sulfonamides, zidovudine, lorazepam, coumadin, sulfamethoxazole, and trimethoprim
• Patients taking Lithium within 30 days of or during this trial
• Incapable of providing informed consent and complying with trial procedures

Where it is running

• Weill Cornell Medical Center/New York Presbyterian Hospital — New York, New York, United States
• Methodist Neurological Institute — Houston, Texas, United States
• Universitäts- und Rehabilitationskliniken Ulm — Ulm, Germany
• Milano Neurological Institute — Milan, Italy
• Umea University — Umeå, Sweden

Full record on ClinicalTrials.gov

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