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Nilotinib for Mild to Moderate Alzheimer's Disease

Active, not recruiting

This study will test nilotinib (Tasigna®), a drug approved to treat chronic myeloid leukemia, a type of blood cancer, in people with mild to moderate Alzheimer's disease. The drug is thought to inhibit a specific molecular pathway that affects Alzheimer's biomarkers.

Minimum Age Maximum Age Gender Healthy Volunteers
50 Years 85 Years All No
January 2017
February 2020

  • Diagnosis of mild to moderate Alzheimer's disease
  • Neuroimaging (MRI or CT scan) result consistent with Alzheimer's within the past year; Mini-Mental State Examination score of 17-24 inclusive
  • Able to take oral medications
  • Caregiver or study partner who has direct contact more than 2 days a week, can come to all visits, and can supervise participant taking study medication
  • Stable medical conditions for 3 months prior to screening; stable medications for 4 weeks prior to screening
  • Stable use of the following medications for 3 months prior to screening is allowed: cholinesterase inhibitors, memantine, vitamin E, estrogens, aspirin, and cholesterol-lowering agents
  • Minimum of 6 years of education or work history sufficient to exclude mental retardation
  • Fluent in English

  • Non-Alzheimer's dementia, Alzheimer's disease with Down syndrome, or familial Alzheimer's disease
  • History of stroke, epilepsy, focal brain lesion, or head injury with loss of consciousness
  • Major psychiatric disorder, including psychosis, depression, or bipolar disorder
  • Alcohol or substance abuse
  • Sensory impairment that would preclude participation
  • Hypokalemia (low potassium), hypomagnesaemia (low magnesium), or long QT syndrome
  • Medications known to disturb how the heart conducts electricity (prolonged QT interval)
  • History of cardiovascular disease, including heart attack, heart failure, angina, and arrhythmia
  • Prescribed strong CYP3A4 inhibitors or history of liver or pancreatic disease
  • Benign or malignant growths or history of cancer within 5 years prior to screening (treated basal or squamous skin cancer or stable prostate cancer are allowed)
  • Contraindications to MRI or lumbar puncture (e.g., pacemaker or taking blood-thinning medication)
  • Evidence of more than four microhemorrhages and/or hemosiderosis
  • Low level of vitamin B12, unless follow-up labs indicate that it is not physiologically significant
  • Enrollment in another trial investigating experimental drug or therapy for Alzheimer's
  • Pregnancy or possible pregnancy
  • HIV-positive

In this study, participants will be randomly assigned to take either nilotinib or a placebo as a capsule taken once a day for the first 6 months, followed by two capsules a day for the next 6 months. Based on preclinical evidence and previous trials of nilotinib for other neurodegenerative diseases, researchers are testing the drug to determine if it can safely inhibit Abl, a molecular pathway linked to Alzheimer's disease. They will also assess if the drug affects cerebrospinal fluid levels of amyloid-beta and tau, two proteins whose harmful forms are features of Alzheimer's, and if the drug can penetrate the blood-brain barrier.

Name City State Zip Status Primary Contact
Georgetown University Medical Center
Washington District of Columbia 20007

Georgetown University

Name Role Affiliation
Raymond S. Turner, MD, PhD Principal Investigator Georgetown University
Charbel E. Moussa, MD, PhD Study Director Georgetown University

Name Phone Email
Raymond S. Turner, MD, PhD 202-687-7337
Ashot R. Shekoyan, MD, PhD 202-687-7591


A Randomized, Double Blind, Placebo-controlled Study to Evaluate the Impact of Low Doses of Nilotinib (Tasigna) on Safety, Biomarkers and Clinical Outcomes in Subjects With Mild to Moderate Alzheimer's Disease