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Funded Studies

Phase IIa Safety and Tolerability Trial of Nilotinib in People with Parkinson's Disease

Study Rationale:
Previous research has shown higher levels of the c-Abl protein are activated in the brains of people with Parkinson's disease (PD), and studies have linked c-Abl to pathways associated with the disease. Impeding the activity of this protein could potentially slow or stop the progression of PD, making it an emerging therapeutic target. While much work remains to understand the role of this protein in Parkinson's, it showed early promise as a potential therapeutic target. A small open-label Phase I clinical trial evaluated the safety and tolerability of nilotinib, a c-Abl inhibitor, in people with advanced Parkinson's and preliminary data showed potential benefit.

The Phase IIa trial aims to expand on 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).

Study Design:
The Michael J. Fox Foundation is launching a multicenter, randomized, double-blind, placebo-controlled Phase IIa clinical trial to learn more about nilotinib's safety in and impact on Parkinson's disease. The trial will be led by Tanya Simuni, MD, chief of movement disorders at Northwestern University Feinberg School of Medicine, and will be carried out at clinical sites across the United States through the Parkinson Study Group, the largest not-for-profit scientific network of Parkinson centers in North America.

In the trial, 75 people with moderate to advanced PD will be randomized to receive 150 mg of nilotinib, 300 mg of nilotinib or placebo daily for six months. Neither patients nor clinicians will know what therapy each person receives until the end of the trial. After six months, all treatments will be stopped and patients will be monitored for another 8 weeks. Results will be analyzed to determine if the drug is safe and tolerable, and which of the drug doses appears to be the maximum tolerated.

If outcomes provide conclusive safety data, a second funded trial will test the highest tolerated dose of nilotinib vs. placebo, again for safety and tolerability, in 60 people with early-stage Parkinson's. Neither patients nor investigators will be aware of which treatment is being administered. After 12 months of daily dosing, therapy will be stopped and participants will be monitored for an additional 10 weeks.

Study leadership is working to secure in-kind donation of drug and placebo for use in the trial.

Additional Support:
MJFF has collaborated with Van Andel Research Institute (VARI) and The Cure Parkinson's Trust (CPT) on the clinical development of nilotinib toward the 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 and The Parkinson Alliance.


  • Tanya Simuni, MD, FAAN

    Chicago, IL United States

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