Nilotinib in Parkinson’s Disease (NILO-PD)

No Longer Recruiting
Northwestern University / Michael J Fox Foundation (MJFF)
Study Design: 
Out of 75 participants that will be recruited and randomly assigned 1:1:1 to the 3 groups (arms), 150mg: 300mg: placebo; all once daily. Intervention duration: 6 months and 2 months follow up (NCT03205488) and 12 months and 3 months follow up (NCT02954978).
PRIMARY OUTCOME MEASURE: Safety and tolerability (both studies). SECONDARY OUTCOME MEASURES: Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) part III (motor function) will be evaluated to determine if Nilotinib has the potential to reduce the MDS-UPDRS motor score as well as a spectrum of motor and non-motor outcome scales, PK profile in cerebrospinal fluid and a number of exploratory biomarkers.
Nilotinib is a drug approved for treatment of chronic leukaemia and as such has a number of safety concerns. The current studies aim to assess safety/ tolerability of nilotinib as well as explore signals of efficacy to determine if the future studies are warranted. There are other molecules targeting the c-ABL pathway in development with potentially better safety profile and better CNS penetration, (Sun Pharma, NCT02970019).