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. |
ClinicalTrials.gov Identifier: NCT00992771 |
Recruitment Status
:
Completed
First Posted
: October 9, 2009
Last Update Posted
: June 18, 2012
|
- Study Details
- Tabular View
- Results Submitted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Spinocerebellar Ataxia Type 3 | Drug: varenicline Drug: placebo | Phase 2 |
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
|
- Changes in the patient's SARA Rating Scale total score [ Time Frame: 25 weeks ]
- Frequency and severity of dose-limiting adverse events [ Time Frame: 25 weeks ]
- The effect of varenicline on quality of life in patients with spinocerebellar ataxia [ Time Frame: 25 weeks ]
- The effect of varenicline on depression and anxiety ratings [ Time Frame: 25 weeks ]
- The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia [ Time Frame: 25 weeks ]

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.
Ages Eligible for Study: | 18 Years to 80 Years (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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).
- Age 18 years to 80 years.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- 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).
- Stable doses of all medications for 30 days prior to study entry and for the duration of the study.
- Ability to ambulate with or without assistance.
- Score of 10 or higher (worse) on the SARA total score.
- Score of 3 or higher (worse) on the 'gait' subsection of the SARA rating scale.
Exclusion Criteria:
- 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).
- Pregnancy or lactation.
- Concurrent participation in another clinical study.
- Patients with a history of substance abuse.
- Patients who currently smoke or have smoked within the past 12 months.
- Presence of psychosis, bipolar disorder, untreated depression (BDI greater than or equal to 21), or history of suicide attempt.
- Concurrent treatment with any MAOIs, Wellbutrin, or nicotine patches.
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 24).
- Legal incapacity or limited legal capacity.
- Presence of severe renal disease (BUN 50% greater than normal or creatinine clearance <60 mL/min) or hepatic disease.
- 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).
- Use of varenicline within the previous 30 days.
- 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).

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 ClinicalTrials.gov identifier (NCT number): NCT00992771
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 |
Principal Investigator: | Theresa Zesiewicz, MD | University of South Florida |
Responsible Party: | Theresa Zesiewicz, Professor of Neurology, University of South Florida |
ClinicalTrials.gov Identifier: | NCT00992771 History of Changes |
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:
Spinocerebellar Ataxias Spinocerebellar Degenerations Ataxia Cerebellar Ataxia Machado-Joseph Disease Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Cerebellar Diseases Brain Diseases Central Nervous System Diseases |
Spinal Cord Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Varenicline Nicotinic Agonists Cholinergic Agonists Cholinergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs |