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

This study has been terminated.
(The DSMB recommended that the study be stopped as a result of concerns regarding safety and intolerability and insufficient evidence of efficacy.)
Sponsor:
Collaborators:
Friedreich's Ataxia Research Alliance
Pfizer
Information provided by (Responsible Party):
University of South Florida
ClinicalTrials.gov Identifier:
NCT00803868
First received: December 4, 2008
Last updated: July 23, 2012
Last verified: July 2012

December 4, 2008
July 23, 2012
May 2009
May 2010   (final data collection date for primary outcome measure)
Friedreich Ataxia Rating Scale (FARS) [ Time Frame: 9 weeks ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00803868 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia
Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

The purpose of this study is to determine if varenicline is effective in treating symptoms of Friedreich's ataxia.

Not Provided
Interventional
Phase 2
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Friedreich's Ataxia
  • Drug: varenicline
    up to 1mg po bid for 9 weeks
  • Drug: placebo
    placebo matching study drug up to 1mg po bid
  • Experimental: 1
    Varenicline
    Intervention: Drug: varenicline
  • Placebo Comparator: 2
    Placebo
    Intervention: Drug: placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
28
May 2010
May 2010   (final data collection date for primary outcome measure)

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.

Exclusion Criteria:

  1. Any unstable illness that in the investigator's opinion preclude participation in this study.
  2. Use of another investigational product within the past 28 days.
  3. Patients with a history of substance abuse.
  4. Presence of preexisting psychiatric illness (specifically schizophrenia, bipolar disorder, or history of suicide attempt).
  5. Presence of diabetes (as determined by fasting blood glucose labs within the past 6 months).
  6. Presence of clinically significant cardiac disease (as determined by the investigator based on EKG and echocardiogram results within the past 6 months). Specifically, patients with an ejection fraction <40% or a prolonged QT interval (>50% of cycle duration) will be excluded. If abnormalities are noted on the EKG or echocardiogram, the patient will be eligible IF they provide clearance from a cardiologist.
  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).
Both
18 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00803868
6
Yes
University of South Florida
University of South Florida
  • Friedreich's Ataxia Research Alliance
  • Pfizer
Principal Investigator: Theresa Zesiewicz, M.D. University of South Florida
University of South Florida
July 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP