Abstract Details

Title Long-term Safety and Clinical Effects of Nilotinib in Parkinson’s Disease

Topic Movement Disorders

Presentation(s) Movement Disorders Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 126

Objective Nilotinib is FDA-approved for leukemia and this open label study investigated the safety, tolerability and potential clinical effects of nilotinib in medically optimized Parkinson’s disease patients.

Background Safety and tolerability were primary objectives and clinical outcomes were exploratory.

Design/Methods Sixty-three patients completed a 15-month Phase 2, double-blind, placebo-controlled study and were re-randomized 1:1 into an open label study of nilotinib, 150mg versus 300mg, for 12 months.

Results Nilotinib was safe and tolerated, no adverse effects seemed to be related to the drug and no differences in adverse events were observed between groups. Exploratory clinical outcomes showed that nilotinib, 300mg, was remarkably stable from baseline to 27 months using partial and total UPDRS. Nilotinib, 150mg versus 300mg, significantly declined using partial or the sum of UPDRS I and II. There was no significant difference in nilotinib, 150mg versus 300mg, using UPDRS III (ON Levodopa) and total UPDRS I-III. Sub-group analysis showed that late start nilotinib, 150mg, significantly worsened using the sum of UPDRS II+III and total UPDRS I-III compared to late start nilotinib, 300mg. Quality of life using PDQ-39 in nilotinib, 150 mg, significantly declined between 15 and 27 months compared to nilotinib, 300 mg, and there was no change in cognition using MoCA between groups.

Conclusions This study provides evidence that nilotinib is safe and tolerated in Parkinson’s disease. The exploratory clinical data will inform an adequately powered larger study to evaluate the efficacy of nilotinib, 300mg, in PD.

Authors/Disclosures
Fernando L. Pagan, MD, F�鶹��ýӳ�� (Georgetown University Hospital Dept of Neurology) PRESENTER	Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Acorda. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Adamas. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amneal. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Merz. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Kyowa Kirin. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sunovion. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Teva. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for US World Meds. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Supernus. Dr. Pagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abbvie. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Acorda. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Adamas. Dr. Pagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amneal. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sunovion. Dr. Pagan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Teva. Dr. Pagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Neurocrine. Dr. Pagan has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Sunovion. Dr. Pagan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Supernus. Dr. Pagan has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Journal of Clinical Parkinsonism and related Disorders. Dr. Pagan has stock in Keiferx. The institution of Dr. Pagan has received research support from US World Meds. The institution of Dr. Pagan has received research support from Novartis.
Yasar A. Torres-Yaghi, MD	Dr. Torres-Yaghi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Acorda. Dr. Torres-Yaghi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Abbvie. Dr. Torres-Yaghi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Amneal. Dr. Torres-Yaghi has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Abbott. Dr. Torres-Yaghi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Kyowa Kirin. Dr. Torres-Yaghi has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Teva. Dr. Torres-Yaghi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sunovion .
	No disclosure on file
	No disclosure on file
Charbel E. Moussa, PhD, MB (Georgetown University)	Dr. Moussa has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for KeifeRX LLC. Dr. Moussa has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sun Pharmaceuticals Research Industry. Dr. Moussa has received stock or an ownership interest from Orphan Therapeutics. The institution of Dr. Moussa has received research support from NIA. The institution of Dr. Moussa has received research support from Sun Pharmaceuticals Research Industry. The institution of Dr. Moussa has received research support from Alzheimer's Association . Dr. Moussa has received intellectual property interests from a discovery or technology relating to health care. Dr. Moussa has received intellectual property interests from a discovery or technology relating to health care. Dr. Moussa has received publishing royalties from a publication relating to health care. Dr. Moussa has received personal compensation in the range of $500-$4,999 for serving as a Grant reviewer with DG Information Technology. Dr. Moussa has received personal compensation in the range of $0-$499 for serving as a Peer Reviews with National Institutes of Health.

�鶹��ýӳ��

�鶹��ýӳ��