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
  Purpose

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


Condition Intervention Phase
Friedreich's Ataxia
Drug: varenicline
Drug: placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

Resource links provided by NLM:


Further study details as provided by University of South Florida:

Primary Outcome Measures:
  • Friedreich Ataxia Rating Scale (FARS) [ Time Frame: 9 weeks ] [ Designated as safety issue: No ]

Enrollment: 28
Study Start Date: May 2009
Study Completion Date: May 2010
Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Varenicline
Drug: varenicline
up to 1mg po bid for 9 weeks
Placebo Comparator: 2
Placebo
Drug: placebo
placebo matching study drug up to 1mg po bid

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00803868

Locations
United States, Florida
University of South Florida
Tampa, Florida, United States, 33612
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
University of South Florida
Friedreich's Ataxia Research Alliance
Pfizer
Investigators
Principal Investigator: Theresa Zesiewicz, M.D. University of South Florida
  More Information

No publications provided

Responsible Party: University of South Florida
ClinicalTrials.gov Identifier: NCT00803868     History of Changes
Other Study ID Numbers: 6
Study First Received: December 4, 2008
Last Updated: July 23, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Varenicline
Nicotinic Agonists
Cholinergic Agonists
Cholinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on October 19, 2014