Nilotinib in Parkinson's Disease

Phase 2

Completed

• Conditions: Parkinson Disease
• Interventions: Drug: Placebo

• Registration Number: NCT03205488
• Lead Sponsor: Northwestern University

Brief Summary

This study will assess the safety and tolerability of daily oral administration of nilotinib (150-300mg once daily) in Parkinson's Disease.

Detailed Description

The purpose of this study is to determine if nilotinib is safe, if it can be tolerated by patients with Parkinson's disease (PD) and to learn if nilotinib has the possibility of effectively treating PD symptoms. Nilotinib has been approved by the Food and Drug Administration (FDA) to treat certain types of cancer (leukemia) but is considered investigational in this study because it has not been approved for treating PD. Twenty-five sites will enroll participants into 2 cohorts,approximately 75 in Cohort 1 and 60 in Cohort 2. Participants with moderate to advanced PD symptoms will be enrolled in Cohort 1, randomly assigned to take nilotinib (150 mg or 300mg) or placebo, and will complete 13 in-person study visits over 8.5 months.

The results from Cohort 1 will determine if either dose of nilotinib (150mg or 300 mg) is safe and tolerable enough to move forward and evaluate in Cohort 2. If either dose is found to be safe and tolerable, participants with early PD will be enrolled into Cohort 2.

Participants in Cohort 2 will be randomly assigned to either nilotinib (dose to be determined from Cohort 1 results) or placebo and will complete 17 in-person visits over 14.5 months. For both cohorts, the study visits will include clinical assessment of motor, neuropsychiatric and cognitive testing as well as collection of blood and cerebral spinal fluid, collected by lumbar puncture.

This study will also evaluate if nilotinib can help improve motor symptoms associated with PD. All participants in Cohort 1 and participants in Cohort 2 who have started PD medications will have an assessment of the motor exam (Part III) in a practically defined OFF state (12 hours post dose) and ON state (at least one-hour post dose).

Study Timeline

• Study First Submitted: 2017-06-28
• Study First Submitted QC: 2017-06-29
• Study First Posted: 2017-07-02
• Study Start: 2017-10-16
• Primary Completion: 2019-08-26
• Study Completion: 2019-09-28
• Results First Submitted: 2020-05-28
• Status Verified: 2020-07
• Results First Submitted QC: 2020-07-21
• Last Update Submitted: 2020-07-21
• Results First Posted: 2020-07-22
• Last Update Posted: 2020-07-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 76

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 2	Placebo	Early/de novo Randomized 2:1
Cohort 1	Placebo	Moderate to Advanced PD Population Randomized 1:1:1

Primary Outcome Measures

Name	Time	Method
Tolerability of Nilotinib Over Placebo	6 months	The count of study participants who completed the 6-month study treatment period while active on their original assigned dose
Safety of Nilotinib	We assessed adverse events that were collected from the first dose of study drug until 60 days after the participant's last dose.	The count of study participants who experienced any treatment-related SAE in each treatment group

Secondary Outcome Measures

Name	Time	Method
Change in MDS-UPDRS Part III	The MDS-UPDRS Part III ON state was collected at baseline, day 14, day 30, month 3, month 6, 30 and 60 days post treatment. The OFF state was collected at baseline, month 3, month 6, 30 and 60 days post treatment.	The Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III is a motor examination in both practically defined medications OFF state (12 hrs post dose) and ON state (based on the participant/site investigator defined best ON and/or approximately 1 hour post dose). Measure Description: The part III subscale score ranges from 0-165. The Larger the value stands for more disability from PD.

Trial Locations

Locations (24)

Albany Medical College; 🇺🇸 Albany, New York, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Cleveland Clinic - Las Vegas; 🇺🇸 Las Vegas, Nevada, United States

University of Cincinnati; 🇺🇸 Cincinnati, Ohio, United States

John Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Barrow Neurological Institute; 🇺🇸 Sun City, Arizona, United States

Beth Israel Medical Center; 🇺🇸 New York, New York, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States

University of California Davis; 🇺🇸 Sacramento, California, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States

University of Colorado at Denver; 🇺🇸 Aurora, Colorado, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Michigan State University; 🇺🇸 East Lansing, Michigan, United States

Cleveland Clinic; 🇺🇸 Cleveland, Ohio, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Virginia; 🇺🇸 Charlottesville, Virginia, United States

Medical University of South Carolina; 🇺🇸 Charleston, South Carolina, United States

Inland Northwest Research; 🇺🇸 Spokane, Washington, United States