Working… Menu

Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00992771
Recruitment Status : Completed
First Posted : October 9, 2009
Last Update Posted : June 18, 2012
National Ataxia Foundation
Bob Allison Ataxia Research Center (BAARC)
Information provided by (Responsible Party):
Theresa Zesiewicz, University of South Florida

Brief Summary:
Spinocerebellar ataxia (SCA) is a group of inherited disorders characterized by cerebellar degeneration leading to imbalance, incoordination, speech difficulties and problems with walking. Recently, individual case reports have suggested that varenicline, a drug used in smoking cessation, produces substantial improvement in patients with several inherited ataxias. A modest response was noted in 5 patients with SCA, suggesting that it is potentially efficacious in this disorder as well. Although this agent is available for off-label use, the severe side effects noted with its use and the lack of long-term toxicity data demand that it be systematically assessed. The present study will test whether varenicline is safe and potentially efficacious in a heterogeneous cohort of adults with SCA.

Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia Type 3 Drug: varenicline Drug: placebo Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Pilot, Randomized, Double-blind, Placebo-controlled Phase I Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
Study Start Date : October 2009
Actual Primary Completion Date : April 2011
Actual Study Completion Date : April 2011

Arm Intervention/treatment
Experimental: Varneicline Drug: varenicline
up to 1mg BID for 8 weeks

Placebo Comparator: Placebo Drug: placebo
placebo matching varenicline, up to 1mg BID for 8 weeks

Primary Outcome Measures :
  1. Changes in the patient's SARA Rating Scale total score [ Time Frame: 25 weeks ]
  2. Frequency and severity of dose-limiting adverse events [ Time Frame: 25 weeks ]

Secondary Outcome Measures :
  1. The effect of varenicline on quality of life in patients with spinocerebellar ataxia [ Time Frame: 25 weeks ]
  2. The effect of varenicline on depression and anxiety ratings [ Time Frame: 25 weeks ]
  3. The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia [ Time Frame: 25 weeks ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Outpatients with spinocerebellar ataxia type 3 diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient).
  2. Age 18 years to 80 years.
  3. Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
  4. Serum creatine kinase, complete metabolic panel, complete blood count, liver function tests, renal function tests, platelets and EKG are within normal limits (results obtained from primary care physician and dated within the past 6 months or obtained at screening visit).
  5. Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
  6. Ability to ambulate with or without assistance.
  7. Score of 10 or higher (worse) on the SARA total score.
  8. Score of 3 or higher (worse) on the 'gait' subsection of the SARA rating scale.

Exclusion Criteria:

  1. Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
  2. Pregnancy or lactation.
  3. Concurrent participation in another clinical study.
  4. Patients with a history of substance abuse.
  5. Patients who currently smoke or have smoked within the past 12 months.
  6. Presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt.
  7. Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
  8. Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24).
  9. Legal incapacity or limited legal capacity.
  10. Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance <60 mL/min) or hepatic disease.
  11. Abnormal creatine kinase and/or platelet count in the past 6 months (as determined by lab reports obtained from primary care physicians or conducted at baseline).
  12. Use of varenicline within the previous 30 days.
  13. Ataxia derived from any other cause than genetically-confirmed SCA (including but not limited to alcoholism, head injury, Multiple Sclerosis, olivo-ponto-cerebellar atrophy or multiple system atrophy).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00992771

Layout table for location information
United States, California
University of California - Los Angeles
Los Angeles, California, United States, 90095
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
University of South Florida
Tampa, Florida, United States, 33612
Sponsors and Collaborators
University of South Florida
National Ataxia Foundation
Bob Allison Ataxia Research Center (BAARC)
Layout table for investigator information
Principal Investigator: Theresa Zesiewicz, MD University of South Florida
Layout table for additonal information
Responsible Party: Theresa Zesiewicz, Professor of Neurology, University of South Florida Identifier: NCT00992771    
Other Study ID Numbers: 8
First Posted: October 9, 2009    Key Record Dates
Last Update Posted: June 18, 2012
Last Verified: June 2012
Additional relevant MeSH terms:
Layout table for MeSH terms
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Nicotinic Agonists
Cholinergic Agonists
Cholinergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs