Trial Outcomes & Findings for Impact of Nilotinib on Safety, Biomarkers and Clinical Outcomes in Mild to Moderate Alzheimer's Disease (NCT NCT02947893)
NCT ID: NCT02947893
Last Updated: 2026-07-23
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
37 participants
Primary outcome timeframe
12 months
Results posted on
2026-07-23
Participant Flow
Participant milestones
| Measure |
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.
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
|
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.
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
|
|---|---|---|
|
Overall Study
COMPLETED
|
17
|
14
|
|
Overall Study
NOT COMPLETED
|
3
|
3
|
|
Overall Study
STARTED
|
20
|
17
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Impact of Nilotinib on Safety, Biomarkers and Clinical Outcomes in Mild to Moderate Alzheimer's Disease
Baseline characteristics by cohort
| Measure |
Group 1 (Placebo)
n=20 Participants
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.
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
|
Group 2 (Treated)
n=17 Participants
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.
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
|
Total
n=37 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Region of Enrollment
United States
|
20 participants
n=9 Participants
|
17 participants
n=27 Participants
|
37 participants
n=267 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=9 Participants
|
3 Participants
n=27 Participants
|
8 Participants
n=267 Participants
|
|
Age, Categorical
>=65 years
|
15 Participants
n=9 Participants
|
14 Participants
n=27 Participants
|
29 Participants
n=267 Participants
|
|
Age, Continuous
|
69.2 years
STANDARD_DEVIATION 6.6 • n=9 Participants
|
72.2 years
STANDARD_DEVIATION 6.8 • n=27 Participants
|
70.6 years
STANDARD_DEVIATION 6.8 • n=267 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=9 Participants
|
14 Participants
n=27 Participants
|
27 Participants
n=267 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=9 Participants
|
3 Participants
n=27 Participants
|
10 Participants
n=267 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=9 Participants
|
2 Participants
n=27 Participants
|
3 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=267 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=9 Participants
|
15 Participants
n=27 Participants
|
32 Participants
n=267 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=267 Participants
|
PRIMARY outcome
Timeframe: 12 monthsSafety 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.
Outcome measures
| Measure |
Group 1 (Placebo)
n=20 Participants
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.
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
|
Group 2 (Treated)
n=17 Participants
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.
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
|
|---|---|---|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Liver enzyme elevation
|
0 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Lesions
|
10 events
|
4 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Erythema
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Cerumen Impaction
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Belching
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Syncopy
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Hypertension
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Sclera redness
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Blurred vision
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Pinpoint pupils
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Retinal detachment
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Eye injection
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Floaters
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Soreness
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Vomiting
|
3 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Diarrhea
|
3 events
|
7 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Acid reflux
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
GI virus
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Hemorrhoid
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Abdominal pain
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Constipation
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Diverticulitis
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Anemia
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Thrombocytopenia
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Leukopenia
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Lipase elevation
|
0 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Amylase elevation
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Hypercholesterolemia
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Swelling
|
2 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Stiffness
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Pain
|
12 events
|
7 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Tenderness
|
1 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Arthroplasty
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Weakness
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Numbness
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Cramps
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Torn rotator cuff
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Headaches
|
4 events
|
4 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Pot-LP headache
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Dizziness
|
3 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Daytime sleep
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Gait instability
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Lightheadedness
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Aphasia
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Confusion
|
2 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Hallucinations
|
4 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Disinhibition
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Agitation
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Anxiety/Paranoia
|
2 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Mood swings
|
0 events
|
12 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Suicidal ideation
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Incontinence
|
4 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
UTI
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Cough
|
4 events
|
6 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Sinusitis
|
7 events
|
6 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
URI
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Sore throat
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Bronchitis
|
0 events
|
3 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Nodules
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Dermatitis
|
4 events
|
6 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Bruising
|
1 events
|
4 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Lumpectomy
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Candida
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Squamous cell carcinoma
|
0 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Falls
|
1 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Fatigue
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Tingling
|
2 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Allergies
|
1 events
|
1 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Nosebleed
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Dry hair
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Low thyroxine
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Iron sensation in abdomen
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Increased potassium
|
1 events
|
0 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Mouth taste
|
0 events
|
2 events
|
|
Safety Will be Measured by Number of Participants Experiencing the Occurrence of Adverse Events and/or Abnormal Laboratory Values
Weight loss/reduced appetite
|
0 events
|
1 events
|
SECONDARY outcome
Timeframe: 12 monthsTo determine the Cmax (ng/ml), we will measure Bosutinib in both the CSF and Plasma and perform population pharmacokinetics using Liquid chromatography-tandem mass spectrometry (LC-MS/MS)
Outcome measures
| Measure |
Group 1 (Placebo)
n=20 Participants
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.
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
|
Group 2 (Treated)
n=17 Participants
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.
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
|
|---|---|---|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
CSF
|
0 ng/ml
Standard Deviation 0
|
1.2 ng/ml
Standard Deviation 0.74
|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
Plasma
|
0 ng/ml
Standard Deviation 0
|
410.6 ng/ml
Standard Deviation 161.7
|
SECONDARY outcome
Timeframe: 12 monthsTo determine the Tmax, we will measure Bosutinib in both the CSF and Plasma and perform population pharmacokinetics using Liquid chromatography-tandem mass spectrometry (LC-MS/MS)
Outcome measures
| Measure |
Group 1 (Placebo)
n=20 Participants
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.
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
|
Group 2 (Treated)
n=17 Participants
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.
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
|
|---|---|---|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
CSF
|
0 hours
Standard Deviation 0
|
3 hours
Standard Deviation 0
|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
Plasma
|
0 hours
Standard Deviation 0
|
4 hours
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 12 monthsTo determine the AUC (ng/ml\*h) , we will measure Bosutinib in both the CSF and Plasma and perform population pharmacokinetics using Liquid chromatography-tandem mass spectrometry (LC-MS/MS)
Outcome measures
| Measure |
Group 1 (Placebo)
n=20 Participants
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.
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
|
Group 2 (Treated)
n=17 Participants
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.
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
|
|---|---|---|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
Plasma
|
0 ng/ml*h
Standard Deviation 0
|
2507 ng/ml*h
Standard Deviation 0
|
|
Pharmacokinetics of Nilotinib in Individuals With Alzheimer's Disease
CSF
|
0 ng/ml*h
Standard Deviation 0
|
7.59 ng/ml*h
Standard Deviation 0
|
Adverse Events
Group 1 (Placebo)
Serious events: 3 serious events
Other events: 20 other events
Deaths: 0 deaths
Group 2 (Treated)
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group 1 (Placebo)
n=20 participants at risk
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.
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
|
Group 2 (Treated)
n=17 participants at risk
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.
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
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Vertigo
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Psychosis
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Cardiac disorders
Hypotension
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
Other adverse events
| Measure |
Group 1 (Placebo)
n=20 participants at risk
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.
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
|
Group 2 (Treated)
n=17 participants at risk
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.
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
|
|---|---|---|
|
Eye disorders
Soreness
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
2/20 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
2/20 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
35.3%
6/17 • Number of events 7 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Acid reflux
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Hemorrhoids
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
GI virus
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Gastrointestinal disorders
Diverticulitis
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Hepatobiliary disorders
Liver enzyme elevation
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Hepatobiliary disorders
Lipase elevation
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
17.6%
3/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Hepatobiliary disorders
Amylase elevation
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Hepatobiliary disorders
Hypercholesterolemia
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Swelling
|
10.0%
2/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Stiffness
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Pain
|
30.0%
6/20 • Number of events 12 • Adverse events (AEs) were reported over 12 months.
|
29.4%
5/17 • Number of events 7 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Headaches
|
20.0%
4/20 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
17.6%
3/17 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Post-LP headache
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Dizziness
|
15.0%
3/20 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Daytime sleep
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Gait Instability
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Lightheadedness
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Nervous system disorders
Aphasia
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Confusion
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Hallucinations
|
15.0%
3/20 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Disinhibition
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Agitation
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Anxiety/paranoia
|
5.0%
1/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Mood swings
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
47.1%
8/17 • Number of events 12 • Adverse events (AEs) were reported over 12 months.
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Renal and urinary disorders
Incontinence
|
15.0%
3/20 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
17.6%
3/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Renal and urinary disorders
UTI
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.0%
3/20 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
29.4%
5/17 • Number of events 6 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
25.0%
5/20 • Number of events 7 • Adverse events (AEs) were reported over 12 months.
|
23.5%
4/17 • Number of events 6 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
URI
|
10.0%
2/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
17.6%
3/17 • Number of events 3 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Nodules
|
10.0%
2/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Lesions
|
35.0%
7/20 • Number of events 10 • Adverse events (AEs) were reported over 12 months.
|
23.5%
4/17 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
20.0%
4/20 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
23.5%
4/17 • Number of events 6 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Bruising
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
23.5%
4/17 • Number of events 4 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Lumpectomy
|
5.0%
1/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Candida
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Skin and subcutaneous tissue disorders
Squamous cell carcinoma
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Arthroplasty
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Tenderness
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Weakness
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Numbness
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Cramps
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Musculoskeletal and connective tissue disorders
Torn Rotator Cuff
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Eye injection
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Floaters
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Falls
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
11.8%
2/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Fatigue
|
10.0%
2/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Tingling
|
10.0%
2/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Allergies
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Nosebleed
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Dry Hair
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Low thyroxine
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Iron sensation in abdomen
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Increased potassium
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Cerumen Impaction
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Belching
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Mouth taste
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
|
General disorders
Weight loss/reduced appetite
|
0.00%
0/20 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Cardiac disorders
Syncope
|
5.0%
1/20 • Number of events 2 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Cardiac disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Sclera
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Blurred vision
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
5.9%
1/17 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Pinpoint pupils
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
|
Eye disorders
Retinal detachment
|
5.0%
1/20 • Number of events 1 • Adverse events (AEs) were reported over 12 months.
|
0.00%
0/17 • Adverse events (AEs) were reported over 12 months.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place