Leukemia and Parkinson’s disease, like most diseases, ravage the body of all it was capable of doing. The two diseases were never known to be closely correlated until Nilotinib, a drug used to treat chronic myelogenous leukemia patients, was apparently found to ease some of the complications of Parkinson’s.
A small research group at Georgetown University coordinated the original clinical trial, in which the results of Nilotinib showed promising results in all 12 subjects. But because the trial had a small sample size, the University of Iowa is conducting a broader study with the hope of proving the drug is safe for Parkinson’s patients.
“As with any chemotherapy-type drug, there are lots of side effects,” said Dixie Ecklund, the director of operations at the UI Public Health College Clinical Trials Statistical & Data Management Center. “If this drug is proven to be safe and tolerated and then goes on to a larger study to show symptomatic improvements [efficacy] in motor and cognitive functions for Parkinson’s patients, this would provide a new tool for clinicians to help ease the burden on Parkinson’s patients.”
The Michael J. Fox Foundation is interested in the study, and it works with the UI to establish a safe and productive trial to help make the study as efficient as possible. The research should take approximately four years.
The first phase of the study will take 18 months and is to be completed in 2019. The trial will operate as two independent studies.
“The first cohort [which is starting now] will enroll 75 [Parkinson’s] subjects with moderate to late disease,” said UI Professor Chris Coffey, the leader of the study and director of the Clinical Trials Center. “They will be enrolled in a 1:1:1 to receive either placebo, Nilotinib 150 mg once daily, or Nilotinib 300 mg once daily. Each subject will be followed for a period of six months. The primary objective of cohort 1 is to assess the safety and tolerability of the Nilotinib in this moderate/advanced [disease] cohort.”
The data will be collected from 30 sites in the U.S. and be entered in a database created by the trial’s Data Coordinating Center, located at the University of Rochester.
“Our team at Iowa will work together with the [coordinating center] to run summary reports throughout the data-collection process to monitor patient enrollment and to ensure that the data being entered are valid and complete,” said Chelsea Caspell-Garcia, a biostatistician at the UI Clinical Trials Center. “After data collection is complete, our team at Iowa will run statistical analyses to examine the study objectives.”
During the study, the question remains: What defines successful trial and successful results?
“This study will be ‘successful’ if we answer the primary question and definitively show the Nilotinib is or is not safe and tolerable in a [Parkinson’s] cohort,” Coffey said. “The study will be successful from the perspective of [Parkinson’s] patients if we confirm that the drug is safe and tolerable.”
