Nilotinib in Parkinson's Disease

NCT03205488 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: NILO-PD
• Study Type: interventional
• Target: 76
• Locations: 1 country
• Sites: 24

Brief Summary

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

Conditions

Parkinson Disease

Keywords

Northwestern; Nilotinib; USA; NILO-PD; Parkinson Disease

Outcome Measures

Primary Outcomes (2)

• Tolerability of Nilotinib Over Placebo

  The count of study participants who completed the 6-month study treatment period while active on their original assigned dose

  6 months

• Safety of Nilotinib

  The count of study participants who experienced any treatment-related SAE in each treatment group

  We assessed adverse events that were collected from the first dose of study drug until 60 days after the participant's last dose.

Secondary Outcomes (1)

• 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.

Study Arms (2)

Cohort 1

ACTIVE COMPARATOR

Moderate to Advanced PD Population Randomized 1:1:1

Drug: Cohort 1:Nilotinib Oral Capsules (150mg or 300mg); Drug: Placebo

Cohort 2

ACTIVE COMPARATOR

Early/de novo Randomized 2:1

Drug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1); Drug: Placebo

Interventions

Cohort 1:Nilotinib Oral Capsules (150mg or 300mg) DRUG

2 capsules taken once daily

Cohort 1

Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1) DRUG

2 capsules taken once daily

Cohort 2

Placebo DRUG

2 capsules taken once daily

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 40 Years - 79 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Idiopathic PD based on the UK Brain Bank diagnostic criteria.
• Any race and either gender, age 40-79
• Able to read and understand English with the capacity to provide voluntary informed consent by signing the informed consent form (ICF)
• Willing to comply with all study procedures including multiple lumbar punctures (LP)
• Must be on a stable regimen of central nervous system acting medications (if applicable) for at least 30 days prior to the baseline visit (e.g., benzodiazepines, antidepressants, hypnotics)
• a. Diagnosis of PD duration \> 5 year 7a. Hoehn \& Yahr scale (H\&Y) stage \> 2 and \< 4 in the ON state 8a. Must be on a stable regimen of PD medications, that includes levodopa, for at least 30 days prior to the screening visit
• a. Treatment with monoamine oxidase B (MAO-B) inhibitors will be allowed provided the dose has been stable for 60 days prior to baseline
• b. Diagnosis of PD duration \< 3 years 7b. H\&Y stage ≤ 2 8b. Participants who are currently NOT receiving symptomatic therapy (ST) (levodopa,dopamine agonists and monoamine oxidase B (MAO-B) inhibitors) and NOT projected to require ST for at least 3 months from enrollment.
• a. Treatment with amantadine or an anticholinergic agent will be allowed provided the dose has been stable for 30 days prior to screening and will remain stable for the duration of the study

You may not qualify if:

• Diagnosis of atypical parkinsonism
• History of bipolar disorder or major depression, or presence of active depression defined as a Beck Depression Inventory II (BDI-II) score \>17
• History of a suicide attempt within the last 5 years or active suicidal ideations
• History of schizophrenia or schizophrenia spectrum disorders
• History of uncontrolled hypokalemia or hypomagnesaemia, or laboratory evidence of such on screening
• History of cardiac arrhythmia, long QT syndrome, or a corrected QT interval (QTcF) ≥450ms at screening visit 1
• Treated within 30 days prior to randomization, or planned use during the trial with any of the following classes of Concomitant drugs:
• Class IA or III antiarrhythmic drugs
• QT prolonging drugs
• Strong CYP3A4 inhibitors or inducers
• Anticoagulants
• Proton pump inhibitors
• A clinical history, or the active presence of a cardiovascular condition including:
• Myocardial infarction, known cardiac ischemia, or angina
• Cerebrovascular event (e.g. embolic stroke)

Sponsors & Collaborators

• Northwestern University: lead
• University of Rochester: collaborator
• University of Iowa: collaborator
• Michael J. Fox Foundation for Parkinson's Research: collaborator

Study Sites (24)

University of Alabama at Birmingham

Birmingham, Alabama, 35294-0017, United States

Location

Barrow Neurological Institute

Sun City, Arizona, 85013, United States

Location

University of California Davis

Sacramento, California, 95817, United States

Location

University of Colorado at Denver

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32607, United States

Location

University of South Florida

Tampa, Florida, 33620, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

John Hopkins University

Baltimore, Maryland, 21093, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Cleveland Clinic - Las Vegas

Las Vegas, Nevada, 89106, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

Beth Israel Medical Center

New York, New York, 10003, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45219, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Inland Northwest Research

Spokane, Washington, 99202, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (2)

• Joshi D, Kulkarni M, Parekh P, Shah S, Greig NH, Acharya S. Targeting protein kinases in Parkinson's disease: the emerging role of phytoconstituents. Nutr Neurosci. 2025 Dec;28(12):1532-1563. doi: 10.1080/1028415X.2025.2531356. Epub 2025 Jul 18.

  PMID: 40680102 DERIVED

• Simuni T, Fiske B, Merchant K, Coffey CS, Klingner E, Caspell-Garcia C, Lafontant DE, Matthews H, Wyse RK, Brundin P, Simon DK, Schwarzschild M, Weiner D, Adams J, Venuto C, Dawson TM, Baker L, Kostrzebski M, Ward T, Rafaloff G; Parkinson Study Group NILO-PD Investigators and Collaborators. Efficacy of Nilotinib in Patients With Moderately Advanced Parkinson Disease: A Randomized Clinical Trial. JAMA Neurol. 2021 Mar 1;78(3):312-320. doi: 10.1001/jamaneurol.2020.4725.

  PMID: 33315105 DERIVED

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Results Point of Contact

• Title: Dr. Tanya Simuni
• Organization: Northwestern University

Tatyana.Simuni@nm.org

312-503-2970

Study Officials

• Tanya Simuni, MD

  Northwestern University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Parkinson's disease and Movement Disorders Center Northwestern University Feinberg School of Medicine

Study Record Dates

• First Submitted: June 28, 2017
• First Posted: July 2, 2017
• Study Start: October 16, 2017
• Primary Completion: August 26, 2019
• Study Completion: September 28, 2019
• Last Updated: July 22, 2020
• Results First Posted: July 22, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (24)