Abstract Details

Title An Open Label Clinical Pilot Study of Resveratrol as a Treatment for Friedreich Ataxia

Topic Movement Disorders

Presentation(s) S43 - (-)

Poster/Presentation
Number 006

Background FRDA is due to a triplet repeat expansion in the FXN gene, resulting in deficiency of the mitochondrial protein frataxin. Resveratrol is a plant-derived compound. It was identified to increase frataxin expression in cellular and mouse models of FRDA, and has anti-oxidant properties.

Objective

Design/Methods 27 participants were enrolled in this open-label sequential clinical pilot study. Thirteen participants received resveratrol 1g daily (low-dose), and fourteen received resveratrol 5g daily (high-dose).

Results 24 participants completed the study (twelve in each dosage group). Preliminary assessment of selected secondary outcome measures has been completed. Improvement in ataxia was evident in the high-dose group (change in ICARS, International Cooperative Ataxia Rating Scale -1.9 points, 95% CI -3.1, -0.8, p=0.004) but not the low-dose group (change in ICARS -0.3 points, 95% CI -3.2, 2.6, p=NS). A significant decrease in the oxidative stress marker plasma F2 isoprostane levels occurred in the high-dose group (-216.9 pmol/L plasma, 95% CI -301.4, -132.2, p<0.001) but not the low-dose group (-45.2 pmol/L plasma, 95% CI -152.9, 62.6, p=0.38, p=NS). Change in lymphocyte frataxin levels and other secondary outcome measures will be presented. No serious adverse events were recorded. Gastrointestinal side effects were a common dose-related adverse event.

Conclusions Preliminary analysis of results from this open-label trial suggests that treatment with high-dose resveratrol for 12 weeks improves some clinical and biological markers of FRDA. A placebo-controlled study is required to assess whether resveratrol is clinically beneficial in FRDA.

Authors/Disclosures
Eppie Yiu, MD (Royal Children'S Hospital Melbourne) PRESENTER	The institution of Dr. Yiu has received research support from Biogen. The institution of Dr. Yiu has received research support from Roche . The institution of Dr. Yiu has received research support from PTC. The institution of Dr. Yiu has received research support from Pfizer .
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
Monique M. Ryan, MD (Childrens Hospital)	Dr. Ryan has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen . Dr. Ryan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring . Dr. Ryan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Ryan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Biogen . The institution of Dr. Ryan has received research support from Save our Sons Muscular Dystrophy . The institution of Dr. Ryan has received research support from Muscular Dystrophy Australia. Dr. Ryan has received publishing royalties from a publication relating to health care.
	No disclosure on file
	No disclosure on file

��ɫ��

��ɫ��