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Could a leukemia drug slow Alzheimer's? small study tests safety
NCT ID NCT02947893
First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This study tested a low dose of nilotinib, a drug used for leukemia, in 37 people with mild to moderate Alzheimer's disease. The goal was to see if the drug is safe and can reach the brain to affect proteins linked to Alzheimer's, like tau and amyloid. Participants took the drug or a placebo daily for a period, and researchers monitored side effects and changes in spinal fluid and brain scans. The study was small and focused on safety, so it does not prove the drug works for Alzheimer's.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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37 people
The number who actually took part.
- Started
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Jan 2017
- Finished
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Mar 2021
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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50 to 85 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Age ≥ 50 2. Fluent in English 3. Biomarker confirmed AD with CSF level of Abeta42 \<600ng/mL 4. Able to ingest oral medications 5. Diagnosis of mild to moderate AD according to dementia criteria outlined by McKhann et al. 6. Neuroimaging (MRI or CT) consistent with the diagnosis of AD within the past year 7. MMSE between 17 and 24 (inclusive) at screening 8. Modified Hachinski score ≤ 4 9. QTc interval 350-460ms, inclusive 10. Caregiver/study partner to accompany participant to all visits and have direct contact with the participant \> 2 days/week 11. Written informed consent 12. Capability and willingness to comply with all study criteria 13. Supervision available for study medication 14. Stable medical conditions for 3 months prior to screening visit 15. Stable medications for 4 weeks prior to screening visit 16. Able to complete baseline assessments 17. Minimum of 6 years of education, or work history sufficient to exclude mental retardation 18. Stable use of cholinesterase inhibitors and memantine (U.S. FDA-approved medications for patients with probable AD), vitamin E (up to 400 IU daily), estrogens, aspirin (81-300 mg daily), and cholesterol-lowering agents for 3 months prior to screening is allowed. 19. Clinical laboratory values within normal limits or, if abnormal, must be judged to be clinically insignificant by the investigator Exclusion Criteria: 1. Non-AD dementia, probable AD with Down syndrome, APP, PS-1, or PS-2 mutations (known familial AD), LBD and Fronto-temporal dementia (FTD) 2. History of clinically significant stroke 3. Current evidence or history in past two years of epilepsy, focal brain lesion, head injury with loss of consciousness or DSM-IV criteria for any major psychiatric disorder including psychosis, major depression, bipolar disorder, alcohol or substance abuse 4. Sensory impairment that would preclude participation/cooperation with the protocol 5. Patients with hypokalemia, hypomagnesaemia, or long QTc syndrome. 6. Concomitant drugs known to prolong the QTc interval (\>461ms) and history of cardiovascular disease, including myocardial infarction or cardiac failure, angina, arrhythmia 7. Prescribed strong CYP3A4 inhibitors or a medical history of liver or pancreatic disease 8. Evidence of any significant clinical disorder or laboratory finding that renders the participant unsuitable for receiving an investigational drug including clinically significant or unstable hematologic, hepatic, cardiovascular, pulmonary, gastrointestinal, endocrine, metabolic, renal or other systemic disease or laboratory abnormality 9. Active neoplastic disease, history of cancer five years prior to screening, including breast cancer (history of treated basal or squamous skin cancer, or stable prostate cancer are not exclusionary) 10. Pregnancy or possible pregnancy 11. Contraindications to LP: prior lumbosacral spine surgery, severe degenerative joint disease or deformity of the spine, platelets \< 100,000, use of Coumadin/warfarin, or history of a bleeding disorder 12. Contraindication to MRI 13. Evidence of more than 4 micro hemorrhages and/or hemosiderosis by a recent (12 months) and/or the screening MRI. 14. A low B12 is exclusionary, unless follow-up labs (homocysteine (HC) and methylmalonic acid (MMA)) indicate that it is not physiologically significant. 15. Enrolled in another active trial investigating an experimental drug or therapy for AD 16. HIV positive
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Georgetown University Medical Center
Washington D.C., District of Columbia, 20057, United States
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Other studies related to the condition(s) this trial covers.
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