Abstract Details

Title Nilotinib is reasonably safe and may halt the disease progression in moderately severe Parkinson’s disease patients

Topic Movement Disorders

Presentation(s) P9 - Poster Session 9 (12:00 PM-1:00 PM)

Poster/Presentation
Number 3-017

Objective The primary objective was nilotinib safety and tolerability in moderately severe PD patients. An exploratory objective is to assess nilotinib effects on motor and non-motor symptoms at baseline, 6, 12 and 15 months.

Background This is a phase II, randomized, double-blind placebo-controlled trial to evaluate the effects of a potential disease modifying drug in PD.

Design/Methods Participants were confirmed to have PD according to the UK Brain Bank diagnostic criteria, with Hoehn and Yahr stage 2.5-3, MDS-UPDRS-III motor score 20-40 and MoCA ≥22. 75 participants were randomized 1:1:1 into placebo, 150mg and 300mg nilotinib oral, once daily for 12 months.

Results Niltonib was found to be reasonably safe with no suspected drug related adverse effects. There was no QTc prolongation or myelosuppression. No differences were observed in MDS-UPDRS-I between all study groups tested by a single rater. All groups showed a significant decline in MDS-UPDRS-II scores between baseline and 12 months and after washout. All groups slightly improved on MDS-UPDRS-III motor between baseline and 6 months. The placebo and the 300 mg nilotinib groups remained stable at 12 months and after washout but there was a significant improvement in MDS-UPDRS-III score between baseline and 15 months in the 150 mg nilotinib group (-2.82 points, 95% CI, -4.75- -0.89). No significant differences between each visit were observed in MDS-UPDRS-IV. No statistically significant differences in MDS-UPDRS measurements were observed between groups. There were no statistically significant differences in PDQ-39 between groups, but the placebo group significantly deteriorated after 6 months, while the nilotinib groups did not change.

Conclusions In conclusion these exploratory measures show no clinical worsening in MDS-UPDRS in the nilotinib groups compared to the placebo group that worsened after 6 months of treatment. Our results will guide the future development of a definitive phase III study to evaluate the effects of nilotinib 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.
	No disclosure on file
	No disclosure on file
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 .
Abigail C. Lawler, MD (Inova neurology)	No disclosure on file
Elizabeth E. Mundel, MD (MedStar Georgetown University Hospital)	No disclosure on file
Nadia Yusuf, MD	No disclosure on file
James N. Starr, DO (NGPG Neurology)	No disclosure on file
Muhammad W. Anjum, MD (LSUHSC-S Department of Neurology)	Dr. Anjum has nothing to disclose.
Shahnaz Miri, MD (Wilmer Eye Institute, Johns Hopkins University)	Dr. Miri has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Lawfirm. Dr. Miri has or had stock in IATRION.Dr. Miri has received intellectual property interests from a discovery or technology relating to health care.
Steven W. Nakano, MD	Dr. Nakano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Kyowa Kirin Group. Dr. Nakano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Acadia Pharmaceuticals.
Amelia Carwin, MD (SCL Health)	Dr. Carwin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acadia. Dr. Carwin has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kyowa Kirin.
	No disclosure on file
	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.

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