USF Health will be the lead site for a new study testing the safety and effectiveness of a potent antioxidant for patients with Friedreich’s ataxia (FA), a life-shortening, degenerative neuromuscular disorder.
The news was greeted with enthusiasm and rounds of applause when announced Thursday evening at the 4th annual scientific symposium “Understanding a Cure,” hosted by the Friedreich’s Ataxia Research Alliance (FARA) and the USF Ataxia Research Center (ARC).
Logistics are still being finalized for the double-blind, placebo-controlled trial sponsored by Edison Pharmaceuticals Inc. The study will primarily measure the effect of Edison’s investigative drug, known as EPI 743, on vision, including contrast and acuity, in FA patients, many of whom experience varying degrees of visual changes. Researchers plan secondarily to evaluate neurologic function using the Functional Assessment Rating Scale.
Intended to begin by winter 2012, the USF-led study is not currently recruiting participants, but patients with a genetically-confirmed FA diagnosis can register on the FARA Patient Registry, which will connect eligible patients with researchers conducting the trial once recruitment begins.