Research Award

Theresa A. Zesiewicz, MD

University of South Florida, Tampa, Florida
A Pilot, Randomized, Double-blind, Placebo-controlled Phase I Study to Determine the Safety and Tolerability of a compound in Treating Spinocerebellar Ataxia Types 3.

This will be a pilot, multi-site, prospective, double-blind, randomized, placebo-controlled trial to examine the safety and tolerability of a compound in treating spinocerebellar ataxia types 3. This study will also assess the effectiveness of this compound on gait and imbalance, quality of life; and activities of daily living (ADL) in patients with spinocerebellar ataxia. The study will have two phases. During the first phase, patients will be randomized to take the compound or placebo for eight weeks. For the second phase, patients will switch and take either the compound or the placebo, whichever they did not take during the first phase. Neither the patient nor the study personnel will know when the patient is taking the real medicine.