Skip to main content

Enrollment Now Open for The Michael J. Fox Foundation Phase IIa Clinical Trial Investigating Nilotinib in Parkinson's Disease

  • Enrollment will open on an ongoing basis at a maximum of 25 clinical sites across the United States
  • Visit for a list of currently recruiting sites and eligibility criteria
  • Patients and clinicians urged to use caution before adding a repurposed therapy such as nilotinib to treatment regimens

NEW YORK -- (November 6, 2017) -- Recruitment opened today for a multicenter, double-blind, randomized, placebo-controlled Phase IIa clinical trial to test safety and tolerability of nilotinib in Parkinson's disease (PD). The trial, funded by The Michael J. Fox Foundation (MJFF), aims to enroll a total of 75 people with moderate to advanced Parkinson's at a maximum of 25 participating clinical sites across the United States. Patients are encouraged to visit for a list of recruiting sites and eligibility criteria.

Trial participants will be randomly assigned to receive daily doses of nilotinib or placebo for six months. Each will be closely monitored throughout the dosing period and for eight weeks after all treatments are stopped to gain insights into nilotinib's safety and tolerability, and to determine the maximum dose tolerated. Volunteers will complete motor, cognitive and biological (i.e., blood and spinal fluid) tests, providing data for investigators to evaluate the potential impact of nilotinib on Parkinson's symptoms and/or disease progression. If outcomes provide conclusive safety data, a second trial will test nilotinib in a group of 60 people with early PD for 12 months at the highest tolerated daily dose of niltotinib versus placebo.

The trial is led by principal investigator Tanya Simuni, MD, professor of neurology and head of the division of movement disorders at Northwestern University Feinberg School of Medicine and carried out through the Parkinson Study Group, the largest not-for-profit scientific network of Parkinson's disease centers in North America.

Investigating Nilotinib as a Repurposed Therapy in Parkinson's
Nilotinib, a U.S. Food and Drug Administration (FDA)-approved treatment for chronic myelogenous leukemia, is one of many examples of a repurposed (or repositioned) drug for Parkinson's. Repurposing is a promising and relatively efficient way to seed the pipeline of drugs by taking an existing medicine approved by the FDA for one condition and using it to treat another. Repurposed drugs can usually skip Phase I clinical testing because they are already proven safe in human volunteers. However, Phase II and Phase III trials are required to rigorously evaluate these treatments in Parkinson's.

"Repurposing is a promising path to new Parkinson's treatments, but we must demonstrate the safety and efficacy of therapies specifically for PD," says Todd Sherer, PhD, CEO at MJFF. "As part of our no-stone-unturned approach, The Michael J. Fox Foundation pursues every avenue, including repurposing, to accelerate breakthroughs for patients who urgently need them."

Patients and clinicians are urged to wait for additional data before adding nilotinib or any other repurposed treatments to their regimen.

Clinical Assessment of Nilotinib in Parkinson's
Nilotinib inhibits the activity of c-Abl, a protein that has been linked to cellular pathways associated with Parkinson's disease. Preliminary data from a small open-label Phase I clinical trial evaluating the safety and tolerability of nilotinib in people with advanced Parkinson's showed potential benefit in PD. The Phase IIa trial aims to expand on these preliminary safety findings, and assess potential dosing range, to better understand the implications of nilotinib's long-term use in Parkinson's. The study also will further explore nilotinib's potential to treat symptoms or to slow or stop disease progression (something no current PD treatment has been proven to do).

Repurposing c-Abl compounds, such as nilotinib, within the context of Parkinson's disease is an area of increasing research focus. The MJFF-funded nilotinib trial joins others currently in the recruiting and planning stages. Multiple concurrent trials such as these can provide more data to speed conclusions about a drug's therapeutic potential.

MJFF has collaborated with Van Andel Research Institute (VARI) of Grand Rapids, Michigan, and The Cure Parkinson's Trust (CPT) of London, United Kingdom, toward the development and launch of this clinical trial. Partial funding for the trial has been generously provided through an anonymous leadership gift from a family living with Parkinson's disease, as well as contributions from the Demoucelle Parkinson Charity (Belgium) and The Parkinson Alliance (Kingston, New Jersey). Novartis Pharmaceuticals Corporation is providing in-kind donation of study drug and placebo for use in the trial.

# # #

Media Contact:
Nyssa Kourakos

About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, industry leaders, government research funders and regulators, clinical trial participants, donors and volunteers. In addition to funding more than $750 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. The Foundation increases the flow of participants into Parkinson's disease clinical trials with its online tools Fox Trial Finder and Fox Insight; sponsors a landmark, international study to find reliable and consistent biomarkers of Parkinson's progression; promotes Parkinson's awareness through high-profile events and outreach; advocates for state and federal policies that support medical research and increased access to health care; and coordinates the involvement of thousands of Team Fox members and grassroots volunteers around the world.

For more information, visit us on the Web, Facebook, Twitter, LinkedIn, Instagram and Pinterest.

We use cookies to ensure that you get the best experience. By continuing to use this website, you indicate that you have read our Terms of Service and Privacy Policy.