Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design

Lisa S Friedman, Jennifer M Farmer, Susan Perlman, George Wilmot, Christopher M Gomez, Khalaf O Bushara, Katherine D Mathews, S H Subramony, Tetsuo Ashizawa, Laura J Balcer, Robert B Wilson, David R Lynch
Movement Disorders: Official Journal of the Movement Disorder Society 2010 March 15, 25 (4): 426-32
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.

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