The Crest-E Study of Creatine in Huntington's Disease

• Activity Number: 48
• Type: Invited
• Date/Time: Sunday, July 31, 2011 : 4:00 PM to 5:50 PM
• Sponsor: WNAR
• Author(s): David Oakes*+
• Companies: University of Rochester
• Keywords: clinical trial ; complementary medicine ; nutritional supplement

Abstract

Huntington's disease is an autosomal dominant inherited neurodegenerative disorder characterized by progressive motor dysfunction, emotional disturbance dementia and weight loss. HD occurs in all races and ethnic groups. There are about 30,000 affected individuals in North America, while another 150,000 have a genetic risk. There is no therapy proven to delay onset or slow progression. Currently classed as a nutritional supplement, creatine is a naturally occurring compound produced in the body or provided externally by dietary meat or fish. Creatine monohydrate is sold as a dietary supplement for the improvement of exercise performance an increase of fat-free mass. The CREST-E study (PI Steven M. Hersch M.D., Ph.D, Massachusetts General Hospital, primary sponsor NIH/NCCAM) is a randomized double-blind study of high dose (up to 40g/d) of creatine monohydrate in HD. The study will enroll 650 subjects in approximately 44 sites in equal proportions to study drug or placebo. Primary efficacy outcome will be the change over three years in the total functional capacity. This talk will discuss the design and conduct of this study with some emphasis on statistical issues.