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

Spinocerebellar Ataxia Type 3 Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00992771
• Other study ID #: 8
• Status: Completed
• Phase: Phase 2
• First received: October 8, 2009
• Last updated: June 15, 2012
• Start date: October 2009
• Est. completion date: April 2011

Study information

• Verified date: June 2012
• Source: University of South Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: April 2011
• Est. primary completion date: April 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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).

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

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

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida
United States	University of California - Los Angeles	Los Angeles	California
United States	University of South Florida	Tampa	Florida

Sponsors (4)

Lead Sponsor	Collaborator
University of South Florida	Bob Allison Ataxia Research Center (BAARC), National Ataxia Foundation, Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in the patient's SARA Rating Scale total score		25 weeks
Primary	Frequency and severity of dose-limiting adverse events		25 weeks
Secondary	The effect of varenicline on quality of life in patients with spinocerebellar ataxia		25 weeks
Secondary	The effect of varenicline on depression and anxiety ratings		25 weeks
Secondary	The effect of varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia		25 weeks