Impact of Nilotinib on Safety, Biomarkers and Clinical Outcomes in Mild to Moderate Alzheimer's Disease (AD)
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02947893 |
Recruitment Status : Unknown
Verified March 2019 by R. Scott Turner, Georgetown University.
Recruitment status was: Active, not recruiting
First Posted : October 28, 2016
Last Update Posted : March 13, 2019
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Alzheimer's Disease | Drug: Placebo Capsule(s) Once a Day by Mouth Drug: Nilotinib Capsule(s) Once a Day by Mouth | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 42 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | January 2017 |
Estimated Primary Completion Date : | December 2019 |
Estimated Study Completion Date : | February 2020 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Group 1 (placebo)
Out of 42 total participants with mild to moderate AD (MMSE=17-24 inclusive) and their study partners that will be recruited and 1:1 randomized, 21 (twenty-one) will be assigned to group 1 and given 1 capsule of a placebo drug by mouth every day for the first 6 months followed by 2 capsules once daily for the subsequent 6 months, every time taken without a meal, for the total duration of the study for 12 months.
|
Drug: Placebo Capsule(s) Once a Day by Mouth
1 capsule of Placebo once a day for 6 months followed by 2 capsules of Placebo for another 6 months
Other Name: Nilotinib in Alzheimer's Disease |
Active Comparator: Group 2 (treated)
Out of 42 total participants with mild to moderate AD (MMSE=17-24 inclusive) and their study partners that will be recruited and 1:1 randomized, 21 (twenty-one) will be assigned to group 2 treated with 1 capsule (150mg Nilotinib) once a day by mouth for the first 6 months followed by dose escalation to 2 capsules (300mg Nilotinib) once daily by mouth for the subsequent 6 months, every time taken without a meal, for the total study duration of 12 months.
|
Drug: Nilotinib Capsule(s) Once a Day by Mouth
1 capsule of Nilotinib 150 mg once a day for 6 months followed by 2 capsules of Nilotinib (150 mg each capsule = 300 mb total) for the subsequent 6 months
Other Name: Nilotinib in Alzheimer's Disease |
- Safety will be measured by number of participants experiencing the occurrence of adverse events and/or abnormal laboratory values [ Time Frame: 12 months ]Safety will be measured by assessing number of participants with abnormal laboratory values, as well as adverse events (AEs) and serious adverse events (SAEs) deemed to be possibly, probably, or definitely related to the study drug.
- Effects of Nilotinib treatment on measurement of Nilotinib in the CSF [ Time Frame: 12 months ]The investigators will determine the effects of Nilotinib treatment on Abl inhibition to demonstrate CNS target engagement

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 50 Years to 85 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age ≥ 50
- Fluent in English
- Biomarker confirmed AD with CSF level of Abeta42 <600ng/mL
- Able to ingest oral medications
- Diagnosis of mild to moderate AD according to dementia criteria outlined by McKhann et al.
- Neuroimaging (MRI or CT) consistent with the diagnosis of AD within the past year
- MMSE between 17 and 24 (inclusive) at screening
- Modified Hachinski score ≤ 4
- QTc interval 350-460ms, inclusive
- Caregiver/study partner to accompany participant to all visits and have direct contact with the participant > 2 days/week
- Written informed consent
- Capability and willingness to comply with all study criteria
- Supervision available for study medication
- Stable medical conditions for 3 months prior to screening visit
- Stable medications for 4 weeks prior to screening visit
- Able to complete baseline assessments
- Minimum of 6 years of education, or work history sufficient to exclude mental retardation
- 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.
- Clinical laboratory values within normal limits or, if abnormal, must be judged to be clinically insignificant by the investigator
Exclusion Criteria:
- Non-AD dementia, probable AD with Down syndrome, APP, PS-1, or PS-2 mutations (known familial AD), LBD and Fronto-temporal dementia (FTD)
- History of clinically significant stroke
- 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
- Sensory impairment that would preclude participation/cooperation with the protocol
- Patients with hypokalemia, hypomagnesaemia, or long QTc syndrome.
- Concomitant drugs known to prolong the QTc interval (>461ms) and history of cardiovascular disease, including myocardial infarction or cardiac failure, angina, arrhythmia
- Prescribed strong CYP3A4 inhibitors or a medical history of liver or pancreatic disease
- 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
- 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)
- Pregnancy or possible pregnancy
- 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
- Contraindication to MRI
- Evidence of more than 4 micro hemorrhages and/or hemosiderosis by a recent (12 months) and/or the screening MRI.
- A low B12 is exclusionary, unless follow-up labs (homocysteine (HC) and methylmalonic acid (MMA)) indicate that it is not physiologically significant.
- Enrolled in another active trial investigating an experimental drug or therapy for AD
- HIV positive

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02947893
United States, District of Columbia | |
Georgetown University Medical Center | |
Washington, District of Columbia, United States, 20007 |
Principal Investigator: | Raymond S. Turner, MD, PhD | Georgetown University | |
Study Director: | Charbel E Moussa, MD, PhD | Georgetown University |
Publications of Results:
Responsible Party: | R. Scott Turner, Director, Memory Disorders Program, Georgetown University |
ClinicalTrials.gov Identifier: | NCT02947893 |
Other Study ID Numbers: |
2016-0315 |
First Posted: | October 28, 2016 Key Record Dates |
Last Update Posted: | March 13, 2019 |
Last Verified: | March 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Nilotinib in Alzheimer's disease |
Alzheimer Disease Dementia Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Tauopathies Neurodegenerative Diseases Neurocognitive Disorders Mental Disorders |