To assess the safety and tolerability of daily oral administration of nilotinib (150-300 mg once daily) compared to placebo in moderate/advanced (cohort 1) and early/de novo (cohort 2) Parkinson’s disease (PD) participants.

Several cell and animal model studies suggest that nilotinib may reduce alpha-synuclein pathology in PD. Nilotinib is FDA approved for certain types of leukemia but not for PD. A small open label clinical study tested the safety/tolerability of nilotinib in PD for the first time and reported that there were positive, though exploratory, signs of efficacy.

NILO-PD is a Phase 2A randomized, double-blind, placebo-controlled, parallel group, two cohort study. Cohort 1 will enroll 75 participants with moderate to advanced PD.  Participants will be randomized 1:1:1 to a once daily dose of nilotinib or placebo (150 mg: 300 mg: placebo) for 6 months.  If one or more doses are determined to be safe in cohort 1, then cohort 2 will enroll 60 de novo PD participants randomized 2:1 to a once daily dose of nilotinib or placebo (the highest tolerated and safe dose from cohort 1: placebo) for 12 months. The primary outcome for both cohorts is safety and tolerability. Secondary and exploratory outcomes include assessment of any symptomatic effect of nilotinib, impact of nilotinib on progression of PD disability (MDS-UPDRS OFF/ON), cognitive function (DRS-2), quality of life, PK profile and a battery of serum and cerebrospinal fluid biomarkers.

The study is conducted at 25 Parkinson Study Group (PSG) sites in the US. Recruitment of cohort 1 started November 2017 and is expected to be completed in late 2018.

This study will provide further information on safety/tolerability, dose selection and biomarkers profile of nilotinib as a potential novel symptomatic and disease-modifying therapy for PD and determine whether it is warranted to proceed with future efficacy studies.