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

Sponsor

University of South Florida (Other)

Overall Status

Completed

CT.gov ID

NCT00992771

Collaborator

National Ataxia Foundation (Other), Bob Allison Ataxia Research Center (BAARC) (Other), Pfizer (Industry)

Enrollment

Locations

Arms

Duration (Months)

6.7

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

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

Study Design

Study Type:

Interventional

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 :

Oct 1, 2009

Actual Primary Completion Date :

Apr 1, 2011

Actual Study Completion Date :

Apr 1, 2011

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Changes in the patient's SARA Rating Scale total score [25 weeks]
Frequency and severity of dose-limiting adverse events [25 weeks]

Secondary Outcome Measures

The effect of varenicline on quality of life in patients with spinocerebellar ataxia [25 weeks]
The effect of varenicline on depression and anxiety ratings [25 weeks]
The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia [25 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California - Los Angeles	Los Angeles	California	United States	90095
2	University of Florida	Gainesville	Florida	United States	32610
3	University of South Florida	Tampa	Florida	United States	33612