Update on the Chantix (Varenicline) trial

Thank you to Mike Fernandes who has been keeping close tabs on the Chantix (Varenicline) trial this summer. Here's what Mike learned:

PROTOCOL SYNOPSIS STUDY TITLE

A Pilot, Randomized, Double-blind, Placebo-controlled Phase I Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Types 1,2,3,and 6

SPONSOR

National Ataxia Foundation; Bobby Allison Ataxia Research Center
(Sites: U of South Florida, U of Chicago, UCLA, Emory, U of Florida, U of Minnesota)

CLINICAL PHASE

2

STUDY RATIONALE

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.

STUDY OBJECTIVE(S)

The primary outcomes will be the changes in the patient’s SARA Rating Scale total score and frequency and severity of dose-limiting adverse events.

The secondary objectives of this study are to assess:

the effect of Varenicline on quality of life in patients with spinocerebellar ataxia

the effect of Varenicline on depression and anxiety ratings

the effect of Varenicline on the activity of daily living (ADL) in patients with spinocerebellar ataxia

TEST ARTICLE

Varenicline

STUDY DESIGN

This is a double-blind, parallel group, randomized, placebo-controlled, crossover pilot study

NUMBER OF SUBJECTS

40 subjects overall

6 sites

STUDY DURATION

175 days(± 3 days) per subject

Furthermore, according to Mike, this trial will not be recruiting for test subjects until this summer. Here are a few links that Mike provided to Dr. Theresa A. Zesiewicz of the University of South Florida who will be conducting the study and the study itself:

http://hsc. usf.edu/NR/ rdonlyres/ E1ABA07F- CA90-4B38- B507-E13BCA3FC29 D/0/ProtocolSynopsis.pdf

http://www.ataxia. org/research/ studies/2009/ naf-research- zesiewicz. aspx

http://health. usf.edu/medicine /neurology/ faculty/zesiewic z.htm

On a separate note, Dr. Perlman prescribed Chantix for Richard some time ago to see if it helped him. However, after taking the drug for two months, we did not note any discernible improvement. Therefore, Dr. Perlman decided to take it off Richard's meds list. She will append Richard's results to the study so that his stint and effort as guinea pig will not be wasted.