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