Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3
Status:
Completed
Trial end date:
2011-04-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
University of South Florida
Collaborators:
Bob Allison Ataxia Research Center (BAARC) National Ataxia Foundation Pfizer