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Parkinson DiseasePhase 2NILO-PDNCT03205488

Nilotinib in Parkinson's Disease

A Phase 2 study testing Cohort 1:Nilotinib Oral Capsules (150mg or 300mg) and Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1) in people with Parkinson Disease, run by Northwestern University at 24 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

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

Who is it for?
Ages 40 to 79 years
Conditions the study is for
Parkinson Disease
What is being tested?
Cohort 1:Nilotinib Oral Capsules (150mg or 300mg) and Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
76 participants
Where
24 sitesUnited States
Study dates
16 Oct 2017to 28 Sep 2019
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 40 to 79 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
  • 6a. 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
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  • Treatment with monoamine oxidase B (MAO-B) inhibitors will be allowed provided the dose has been stable for 60 days prior to baseline
  • 6b. 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.
  • 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

Who cannot join

  • 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
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  • 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)
  • Congestive heart failure, symptomatic first degree atrioventricular (AV) block or PR interval > 220msec and all second and third degree AV block, second- or third-degree atrioventricular block, sick sinus syndrome, or other serious cardiac rhythm disturbances
  • History of Torsade de Pointes
  • Other cardiovascular history that, in the opinion of the Site Investigator, will preclude study participation
  • History of hepatic disease, including abnormal liver function defined as Total Bilirubin > 1.5 times upper limit, Aspartate Aminotransferase (AST) and/or Alanine Aminotransferase (ALT) > 2 times the upper limit of the normal, or coagulopathy with INR > 1.4
  • History of epilepsy or a seizure within the last 6 months
  • Active malignancy, or history of a neoplasm in the prior 5 years (excluding basal/squamous cell carcinoma)
  • Prior history of pancreatitis or total gastrectomy or evidence of abnormal pancreatic function defined as elevated amylase and/or lipase > 2 times upper limit of normal
  • Diagnosis of human immunodeficiency virus (HIV), clinically significant chronic hepatitis such as hepatitis B (HBV) or hepatitis C (HCV), or clinical history or signs of an active infection
  • History of drug or alcohol abuse ≤ 5 years
  • Active medical or psychiatric condition that in the opinion of the Site Investigator should preclude study participation
  • Previous surgical management for PD
  • Participants participating in any drug or device clinical investigation concurrently or within 30 days prior to screening for this study
  • Severe lactose and galactose intolerance
  • Participants with evidence of other significant laboratory abnormalities which in the opinion of the site investigator or clinical monitor should preclude study participation
  • Known hypersensitivity or contraindication to study drugs (nilotinib or matching placebo) or their components.
  • Female participants of child-bearing potential. Female participants must be post-menopausal, post-hysterectomy, or have a documented infertility based on a known medical or surgical condition
  • Participants with a history of bone marrow suppression or evidence of persistent myelosuppression defined as absolute neutrophil count <1.8 X 109/L, significant anemia, or thrombocytopenia defined as platelet count < 100 X 109/L
  • 22a. Diagnosis of dementia based on the clinician's assessment, or a Montreal Cognitive Assessment (MoCA) score < 21 at baseline
  • 22b.MoCA score < 26 at baseline 23b. Treated within 60 days prior to randomization or expected to require treatment within 3 months from randomization with any ST (including levodopa and dopamine agonists )
  • 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

Trial locations and contacts

24 sites in 1 country.

24 sites in 1 country

United States24 sites

  • Albany Medical College

    Albany, New York, 12208

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • University of Colorado at Denver

    Aurora, Colorado, 80045

  • John Hopkins University

    Baltimore, Maryland, 21093

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294-0017

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • University of Virginia

    Charlottesville, Virginia, 22903

  • Rush University Medical Center

    Chicago, Illinois, 60612

  • University of Cincinnati

    Cincinnati, Ohio, 45219

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • Michigan State University

    East Lansing, Michigan, 48824

  • University of Florida

    Gainesville, Florida, 32607

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Cleveland Clinic - Las Vegas

    Las Vegas, Nevada, 89106

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Beth Israel Medical Center

    New York, New York, 10003

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19107

  • Oregon Health and Science University

    Portland, Oregon, 97239

  • University of California Davis

    Sacramento, California, 95817

  • Inland Northwest Research

    Spokane, Washington, 99202

  • Barrow Neurological Institute

    Sun City, Arizona, 85013

  • University of South Florida

    Tampa, Florida, 33620

Other trials in United States

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase IIa, Parallel Group, Two Cohort Study to Define the Safety, Tolerability, Clinical and Exploratory Biological Activity of the Chronic Administration of Nilotinib in Participants With Parkinson's Disease

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

Read the 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).

Being tested
Cohort 1:Nilotinib Oral Capsules (150mg or 300mg), Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1), Placebo
Keywords
Northwestern, Nilotinib, USA, NILO-PD, Parkinson Disease
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

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

    2 capsules taken once daily

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

    2 capsules taken once daily

  • PlaceboDrug

    2 capsules taken once daily

Study groups in NCT03205488
Study groupRoleWhat they receive
Cohort 1Active comparatorModerate to Advanced PD Population Randomized 1:1:1
Receives: Drug: Cohort 1:Nilotinib Oral Capsules (150mg or 300mg); Drug: Placebo
Cohort 2Active comparatorEarly/de novo Randomized 2:1
Receives: Drug: Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1); Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 6 months

  2. Safety of Nilotinib

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

    Measured: 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)

  1. Change in MDS-UPDRS Part III

    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.

    Measured: 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.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Jul 2017
  2. Study start16 Oct 2017
  3. Primary completion26 Aug 2019
  4. Study completion28 Sep 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
76 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03205488
Sponsor's protocol ID
NILO-PD
Acronym
NILO-PD
Status last verified
Jul 2020
First submitted
28 Jun 2017
Last updated
22 Jul 2020
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 40680102

  • 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.

    PubMed 33315105

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cohort 1:Nilotinib Oral Capsules (150mg or 300mg) and Cohort 2: Nilotinib Oral Capsules (dose to be determined from Cohort 1) in people with Parkinson Disease, run by Northwestern University at 24 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 35 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 22 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Jul 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Tolerability of Nilotinib Over Placebo (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03205488, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2020.

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