Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

Inclusion Criteria

• •1. Outpatients with FA diagnosed by confirmed by genetic testing.
• •2. Age 18 years to 80 years.
• •3. Women who are not pregnant or breast feeding, and who do not intend to become pregnant. Women of child-bearing potential must use a reliable method of contraception and must provide a negative urine pregnancy test at entry into the study.
• •4. CBC, CMP, and diabetes lab results not indicative of clinically relevant abnormalities (results within the past 6 months prior to screening). These would include but are not limited to:
• •Electrolytes (Chloride, Sodium, Potassium) within laboratory defined normal limits.
• •Hemoglobin, white cell count, platelet count and fasting glucose within laboratory defined normal limits. Creatinine must be (≤1.5 mg/dl).
• •ALT (6-40 u/l) and AST (10-30 u/l) must be less than 2 times normal limit
• •5. Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
• •6. Patient permission (informed consent).
• •7. Ambulatory status: Half of the enrolled patients must be able to ambulate with or without assistance; half of the enrolled patients must be non-ambulatory.
• •7. Presence of current uncontrolled depression as measured by PHQ9 (criteria for depression include all of the following to be present: At least one of the first two questions on PHQ9 endorsed as positive (little pleasure, feeling depressed) indicating the symptom has been present more than half the time in the past two weeks; Question 10 about difficulty at work or home or getting along with others should be answered at least "somewhat difficult."; and the total score ≥ 10.
• •8. Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
• •9. Patients who currently smoke or have smoked within the past 12 months (Smoking will be defined as having smoked any substance even on a single occasion).
• •10. Dementia or other psychiatric illness that prevents the patient from giving informed consent (MMSE less than 25).
• •11. Legal incapacity or limited legal capacity.
• •12. Presence of severe renal disease (creatinine \>1.6) or hepatic disease (AST or ALT\>2x times normal) (as evidenced by labs reported within the past 6 months).
• •13. Abnormal WBC hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).