Clinical Trial: Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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 Typ
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.
Detailed Summary:
Sponsor: University of South Florida
Current Primary Outcome:
- 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 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome: Same as current
Information By: University of South Florida
Dates:
Date Received: October 8, 2009
Date Started: October 2009
Date Completion:
Last Updated: June 15, 2012
Last Verified: June 2012