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| Name | Class |
|---|---|
| National Institutes of Health (NIH) | NIH |
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Dementia with Lewy Bodies (DLB) is an alphasynucleinopathy and the second most common form of dementia in the elderly. DLB shares striking neuropathological and clinical similarities with both Parkinson's disease (PD) and Alzheimer's disease (AD). Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) is approved by the FDA and is well tolerated for CML treatment at oral doses of 600-800mg daily. The Investigators propose to perform a phase II randomized, double blinded, placebo controlled study to evaluate the impact of Nilotinib in patients with DLB.
A phase II randomized, double blinded, placebo controlled study will be performed to evaluate the impact of Nilotinib (Tasigna®, AMN107, Novartis, Switzerland) on safety, tolerability, pharmacokinetics, pharmacodynamics and clinical outcomes in patients with Dementia with Lewy Bodies. Sixty ( 60) participants will be recruited and randomly assigned 1:1 to placebo (arm 1) or 200 mg Nilotinib (arm 2).This study will be conducted in DLB patients with 2.5≥Hoehn & Yahr≤3 and UPDRS I-III ≤50 and 15≥UPDRS III (motor) ≥40 (Unified Parkinson's Disease Rating Score)and MoCA≥18(Montreal Cognitive Assessment). Eligible participants must be stable on MAO-B inhibitors (Rasageline or Selegeline) for 4 weeks and must not be on ≥800mg Levodopa daily. Participants must be stable on acetylcholinesterase inhibitors and other medications for at least 6 weeks. Participants will be treated for 6 months and monitored every month ( 4 weeks) in a total of 9 visits that include screening , baseline, 1, 2, 3, 4, 5, 6 months follow up and 7 month washout. Blood and cerebrospinal fluid (CSF) will be collected at baseline and at 6 months to determine Nilotinib effects on CSF biomarkers.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Sixty (60) participants will be recruited and randomized into 2 arms (1:1). Thirty (30) patients in arm 1 will receive the matching placebo ("sugar pill") one (1) capsule orally (without food) once daily for 6 months (180 days). |
|
| 200 mg Nilotinib | Active Comparator | Sixty (60) participants will be recruited and randomized into 2 arms (1:1). Thirty (30) patients in arm 2 will receive the 200 mg of Nilotinib one (1) capsule orally (without food) once daily for 6 months (180 days). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo oral capsule | Drug | Thirty (30) patients in arm 1 will receive the matching placebo ("sugar pill") one (1) capsule orally (without food) once daily for 6 months (180 days). |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and Tolerability: Occurrence of Adverse Events (AEs) | The Investigators will determine safety and tolerability using the occurrence of adverse events (AEs) of interest as per Nilotinib Investigator Brochure (IB). | 6 Months |
| Measure | Description | Time Frame |
|---|---|---|
| DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on primary biomarkers | 6 Months |
| The Investigators Will Quantify Amyloid Burden Via Florbetaben PET Scan | Quantification of Standardized Uptake Value Ratio (SUVR) of brain amyloid burden via Florbetaben (Neuraceq, is a diagnostic radiotracera) PET at baseline and 6 months (end of treatment (EOT)). Standardized Uptake Value Ratio (SUVR) is a unitless ratio that measures tracer uptake in a target brain region compared to a reference region. A lower SUVR is generally better in amyloid PET scans, as it indicates a lower density of amyloid plaques in the brain while a higher SUVR indicates a higher burden of amyloid plaque accumulation in the brain. |
| Measure | Description | Time Frame |
|---|---|---|
| Measure the Effects of Nilotinib on Cognition Using the Montreal Cognitive Assessment (MoCA) | The MoCA is designed as a rapid screening instrument for mild cognitive dysfunction. It assesses different cognitive domains, including attention and concentration, executive functions, memory, language, visuo-constructional skills, conceptual thinking, calculations and orientation. Scores range between 0 and 30; a score of 26 or higher is generally considered normal, while lower scores indicate impairment. |
Inclusion Criteria:
Exclusion Criteria:
Patients with hypokalemia, hypomagnesaemia, or long QT syndrome- QTc≥461 ms
Concomitant drugs known to prolong the QTc interval and history of any cardiovascular disease, including myocardial infraction or cardiac failure, angina, arrhythmia
History or presence of cardiac conditions including:
Treatment with any of the following drugs at the time of screening or the preceding 30 days, and/or planned use over the course of the trial:
Abnormal liver function defined as AST and/or ALT > 100% the upper limit of the normal
Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
History of HIV, clinically significant chronic hepatitis, or other active infection
Females must not be lactating, pregnant or with possible pregnancy
Medical history of liver or pancreatic disease
Clinical signs indicating syndromes other than DLB, including, PD, PD with Dementia (PDD), corticobasal degeneration, supranuclear gaze palsy, multiple system atrophy, chronic traumatic encephalopathy, signs of frontal dementia, history of stroke, head injury or encephalitis, cerebellar signs, early severe autonomic involvement, Babinski sign
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
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 skin melanoma or stable prostate cancer are not exclusionary)
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
Must not be on any immunosuppressant medications or IVIG
Must not be enrolled as an active participant in another clinical study
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| Name | Affiliation | Role |
|---|---|---|
| Fernando L Pagan, MD | Georgetown University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| MedStar Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23666528 | Result | Hebron ML, Lonskaya I, Moussa CE. Nilotinib reverses loss of dopamine neurons and improves motor behavior via autophagic degradation of alpha-synuclein in Parkinson's disease models. Hum Mol Genet. 2013 Aug 15;22(16):3315-28. doi: 10.1093/hmg/ddt192. Epub 2013 May 10. |
| Label | URL |
|---|---|
| https://sites.google.com/a/georgetown.edu/moussa-lab/lab-members | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Placebo oral capsule: Twenty-two (22) patients in arm 1 received the matching placebo ("sugar pill"); one (1) capsule orally (without food) once daily for 6 months (180 days). |
| FG001 | 200 mg Nilotinib | Nilotinib Oral Capsule: Twenty-one (21) patients in arm 2 received 200 mg of Nilotinib; one (1) capsule orally (without food) once daily for 6 months (180 days). |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo oral capsule: 22 patients in arm 1 received the matching placebo ("sugar pill"); one (1) capsule orally (without food) once daily for 6 months (180 days). |
| BG001 | 200 mg Nilotinib |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Safety and Tolerability: Occurrence of Adverse Events (AEs) | The Investigators will determine safety and tolerability using the occurrence of adverse events (AEs) of interest as per Nilotinib Investigator Brochure (IB). | Posted | Number | events | 6 Months |
|
Adverse events were collected for 6 months for each participant.
Adverse Event reporting is done by reviewing the systems and capturing events that are adverse. All adverse events are logged and filed by date and severity.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo oral capsule: Twenty-two (22) patients in arm 1 received the matching placebo ("sugar pill"); one (1) capsule orally (without food) once daily for 6 months (180 days). |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Falls | Injury, poisoning and procedural complications | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Fernando Pagan | Georgetown University Hospital | 202-444-6087 | Fernando.L.Pagan@gunet.georgetown.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 23, 2020 | Aug 20, 2025 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D020961 | Lewy Body Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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| ID | Term |
|---|---|
| C498826 | nilotinib |
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Investigators will evaluate the effects of 200 mg of Nilotinib versus matching Placebo taken daily by mouth for 6 Months, followed by 1 Month wash-out period in individuals with DLB.
Sixty (60) participants will be recruited and randomly assigned 1:1 to placebo (arm 1) or 200 mg Nilotinib (arm 2). Participants will be treated for 6 months and monitored every month (4 weeks) in a total of 9 visits that include screening, baseline, 1, 2, 3, 4, 5, 6 months follow up and 7-month washout.
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Participants will be randomized by a Biostatistician by an internet based randomization module into two groups. The researchers include : Primary investigator , Sub-Investigators ,Clinical Coordinators, Nurse Practitioners , and Clinical Reseach unit staff.
|
| Nilotinib Oral Capsule | Drug | Thirty (30) patients in arm 2 will receive the 200 mg of Nilotinib one (1) capsule orally (without food) once daily for 6 months (180 days). |
|
|
| Baseline, 6 Months, and change between baseline and 6 months |
| DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on CSF levels of Homavanillic Acid (HVA) between baseline and 6 months. | Changes from Baseline to 6 months |
| DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on the ratio change of phospho-Tau(181)/Abeta42 (pTau(181)/Ab42) in DLB patients | 6 months |
| Change from Baseline in the Montreal Cognitive Assessment at 6 months |
| Measure the Effects of Nilotinib on Cognition Using the Trail Making Test (TMT) | The Trail Making Test (TMT) is a neuropsychological test of visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy. The test can provide information about visual search speed, scanning, speed of processing, mental flexibility, as well as executive functioning. The time to complete the test is measured in seconds. Lower times indicate better executive function, while higher scores suggest impairment. | Change from Baseline in the Trail Making Test at 6 months |
| Measure the Effects of NIlotinib on Cognition Using the Alzheimer's Disease Assessment Scale - Cognitive (ADAS-cog14). | The 14-item Alzheimer's Disease Assessment Scale-Cognitive subscale (ADAS-Cog14) measures cognitive impairment, with higher scores (0-90) indicating greater disability. | Change from Baseline in the Alzheimer's Disease Assessment Scale - cognitive at 6 months |
| Measure the Effects of Nilotinib on Behavior Using the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale (ADCS-ADL) | ADCS-ADL is an activity of daily living inventory to assess functional performance. Using a structured interview format, study partners are queried as to whether participants attempted each item in the inventory during the prior 4 weeks and their level of performance. The ADCS-ADL includes some items from traditional basic ADL tests as well as instrumental (complex) activities of daily living. It is a 23 item scale that provide a total score from 0-78 with a lower score indicating greater severity. | Change from Baseline in the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale at 6 months |
| Measure the Effects of Nilotinib on Behavior Using the Neuropsychiatric Inventory (NPI) | The Neuropsychiatric Inventory (NPI) is a test widely used, clinician-administered tool that evaluates 12 behavioral domains in dementia patients (e.g., agitation, depression, delusions). Each item is scored by multiplying frequency (1-4) by severity (1-3), resulting in a maximum total score of 144, with higher scores indicating greater neuropsychiatric symptoms, often aligning with disease progression. Minimum Score is 0 (no behavioral symptoms present); maximum Score is 144 (highest severity and frequency across all 12 domains). Higher scores indicate a higher frequency and greater severity of neuropsychiatric symptoms (such as delusions, agitation, or apathy) while lower scores indicate few or no behavioral and psychiatric symptoms. | Change from Baseline in Neuropsychiatric Inventory at 6 months |
| Measure the Effects of Nilotinib on Behavior Using the Clinical Assessment of Fluctuation (CAF) | The CAF consists of seven items of confusional behavior (falls, fluctuation, drowsiness, attention, disorganized thinking, altered level of consciousness, communication), scores for which are summed to provide a severity score for fluctuating confusion ranging from 0 to 21. Higher scores indicate more severe fluctuations while lower scores indicate less severe fluctuations. | Change from Baseline in Clinical Assessment of Fluctuation at 6 months |
| Measure the Effects of Nilotinib on Behavior Using the Irritability-Apathy Scale (IAS) | The IAS measures apathy and irritability in patients with dementia. The IAS is a 14-item self-administered questionnaire collecting information about different aspects of irritability and apathy utilizing a 0-3 scale for each item to indicate severity (0 (absent) to 3 (maximum intensity) per question). Total Range is 0-42; a higher total score indicates more severe symptoms, which are often associated with greater morbidity and worse functional outcomes while a lower score indicates lower severity. | Change from Baseline in Irritability-Apathy Scale at 6 months |
| Measure the Effects of Nilotinib on Behavior Using the Problem Behaviors Assessment Short Form (PBA-s) | PBA-s is a structured interview in which a trained interviewer rates the frequency and severity of neuropsychiatric symptoms through observation and the reporting of the Subject and Study Partner. Symptoms rated include depressed mood, suicidal ideation, anxiety, irritability, angry or aggressive behavior, apathy, perseverative thinking or behavior, obsessive-compulsive behaviors, delusional or paranoid thinking, hallucinations, and disoriented behavior. Each behavioral problem is rated for both severity and frequency on a 0-4- point scale; severity and frequency ratings are then multiplied to provide an overall score for each symptom. Minimum score is 0 (symptom is absent); maximum score is 176 (11 items × maximum severity 4 × maximum frequency 4). Higher scores indicate increased frequency or severity of behavioral issues while lower scores indicate decreased severity and frequency. | Change from Baseline in Problem Behaviors Assessment short form at 6 months |
| Measure the Effects of Nilotinib on Motor Function by Using the Unified Parkinson's Disease Rating Scale (UPDRS)-I-III. | UPDRS-I-III is used to follow the longitudinal course of Parkinson's disease. The UPDRS is made up of these sections: Part I: evaluation of mentation, behavior, and mood. Part II: self-evaluation of the activities of daily life (ADLs) Part III: clinician-scored monitored motor evaluation. Part IV: complications of therapy. Part V: Hoehn and Yahr staging of severity of Parkinson's disease. The minimum possible score on the UPDRS is 0, which indicates no disability or no symptoms of Parkinson's disease. The scale, used to assess severity, typically ranges from 0 to 199 (total) (199 being the maximum) or 0 to 108 (motor section, Part III) (108 being maximum). Higher scores on the Unified Parkinson's Disease Rating Scale (UPDRS) Parts I-III indicate increased severity of disease, greater disability, and more significant impairment, while lower scores signify better function. | Change from Baseline in Unified Parkinson's Disease Rating Scale (UPDRS)-I-III at 6 months |
| Measure the Effects of Nilotinib on Motor Function by Using the Timed-Up-And-Go (TUG). | Timed Up and Go (TUG) is an assessment of mobility, balance, walking ability, and fall risk. It measures the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down. This assessment is measured in seconds. A score of less than 10 seconds is normal, 14-20 seconds indicates a high fall risk or frailty, and over 30 seconds suggests significant mobility impairment. | Change from Baseline in Timed-Up-And-Go at 6 months |
Nilotinib Oral Capsule: 21 patients in arm 2 received the 200 mg of Nilotinib; one (1) capsule orally (without food) once daily for 6 months (180 days).
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Secondary | DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on primary biomarkers | Posted | Mean | Standard Deviation | pg/ml | 6 Months |
|
|
|
| Secondary | The Investigators Will Quantify Amyloid Burden Via Florbetaben PET Scan | Quantification of Standardized Uptake Value Ratio (SUVR) of brain amyloid burden via Florbetaben (Neuraceq, is a diagnostic radiotracera) PET at baseline and 6 months (end of treatment (EOT)). Standardized Uptake Value Ratio (SUVR) is a unitless ratio that measures tracer uptake in a target brain region compared to a reference region. A lower SUVR is generally better in amyloid PET scans, as it indicates a lower density of amyloid plaques in the brain while a higher SUVR indicates a higher burden of amyloid plaque accumulation in the brain. | Posted | Mean | Standard Deviation | unitless ratio | Baseline, 6 Months, and change between baseline and 6 months |
|
|
|
| Secondary | DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on CSF levels of Homavanillic Acid (HVA) between baseline and 6 months. | Posted | Mean | Standard Deviation | nM | Changes from Baseline to 6 months |
|
|
|
| Secondary | DLB Related CSF Biomarkers | Pharmacodynamics: Determine the effects of Nilotinib on the ratio change of phospho-Tau(181)/Abeta42 (pTau(181)/Ab42) in DLB patients | Posted | Mean | Standard Deviation | ratio | 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Cognition Using the Montreal Cognitive Assessment (MoCA) | The MoCA is designed as a rapid screening instrument for mild cognitive dysfunction. It assesses different cognitive domains, including attention and concentration, executive functions, memory, language, visuo-constructional skills, conceptual thinking, calculations and orientation. Scores range between 0 and 30; a score of 26 or higher is generally considered normal, while lower scores indicate impairment. | Changes from Baseline to 6 months | Posted | Mean | Standard Deviation | points on a scale | Change from Baseline in the Montreal Cognitive Assessment at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Cognition Using the Trail Making Test (TMT) | The Trail Making Test (TMT) is a neuropsychological test of visual attention and task switching. It consists of two parts in which the subject is instructed to connect a set of 25 dots as quickly as possible while still maintaining accuracy. The test can provide information about visual search speed, scanning, speed of processing, mental flexibility, as well as executive functioning. The time to complete the test is measured in seconds. Lower times indicate better executive function, while higher scores suggest impairment. | Changes from Baseline to 6 months | Posted | Mean | Standard Deviation | seconds | Change from Baseline in the Trail Making Test at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of NIlotinib on Cognition Using the Alzheimer's Disease Assessment Scale - Cognitive (ADAS-cog14). | The 14-item Alzheimer's Disease Assessment Scale-Cognitive subscale (ADAS-Cog14) measures cognitive impairment, with higher scores (0-90) indicating greater disability. | Changes from Baseline to 6 months | Posted | Mean | Standard Deviation | score on a scale | Change from Baseline in the Alzheimer's Disease Assessment Scale - cognitive at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Behavior Using the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale (ADCS-ADL) | ADCS-ADL is an activity of daily living inventory to assess functional performance. Using a structured interview format, study partners are queried as to whether participants attempted each item in the inventory during the prior 4 weeks and their level of performance. The ADCS-ADL includes some items from traditional basic ADL tests as well as instrumental (complex) activities of daily living. It is a 23 item scale that provide a total score from 0-78 with a lower score indicating greater severity. | Posted | Mean | Standard Deviation | points on a scale | Change from Baseline in the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Behavior Using the Neuropsychiatric Inventory (NPI) | The Neuropsychiatric Inventory (NPI) is a test widely used, clinician-administered tool that evaluates 12 behavioral domains in dementia patients (e.g., agitation, depression, delusions). Each item is scored by multiplying frequency (1-4) by severity (1-3), resulting in a maximum total score of 144, with higher scores indicating greater neuropsychiatric symptoms, often aligning with disease progression. Minimum Score is 0 (no behavioral symptoms present); maximum Score is 144 (highest severity and frequency across all 12 domains). Higher scores indicate a higher frequency and greater severity of neuropsychiatric symptoms (such as delusions, agitation, or apathy) while lower scores indicate few or no behavioral and psychiatric symptoms. | Changes from Baseline to 6 months (NPI Freq*Sev) | Posted | Mean | Standard Deviation | score on a scale | Change from Baseline in Neuropsychiatric Inventory at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Behavior Using the Clinical Assessment of Fluctuation (CAF) | The CAF consists of seven items of confusional behavior (falls, fluctuation, drowsiness, attention, disorganized thinking, altered level of consciousness, communication), scores for which are summed to provide a severity score for fluctuating confusion ranging from 0 to 21. Higher scores indicate more severe fluctuations while lower scores indicate less severe fluctuations. | Changes form Baseline to 6 months (CAF Total) | Posted | Mean | Standard Deviation | score on a scale | Change from Baseline in Clinical Assessment of Fluctuation at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Behavior Using the Irritability-Apathy Scale (IAS) | The IAS measures apathy and irritability in patients with dementia. The IAS is a 14-item self-administered questionnaire collecting information about different aspects of irritability and apathy utilizing a 0-3 scale for each item to indicate severity (0 (absent) to 3 (maximum intensity) per question). Total Range is 0-42; a higher total score indicates more severe symptoms, which are often associated with greater morbidity and worse functional outcomes while a lower score indicates lower severity. | Changes from Baseline to 6 months (Patient Irritability Total) | Posted | Mean | Standard Deviation | points on a scale | Change from Baseline in Irritability-Apathy Scale at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Behavior Using the Problem Behaviors Assessment Short Form (PBA-s) | PBA-s is a structured interview in which a trained interviewer rates the frequency and severity of neuropsychiatric symptoms through observation and the reporting of the Subject and Study Partner. Symptoms rated include depressed mood, suicidal ideation, anxiety, irritability, angry or aggressive behavior, apathy, perseverative thinking or behavior, obsessive-compulsive behaviors, delusional or paranoid thinking, hallucinations, and disoriented behavior. Each behavioral problem is rated for both severity and frequency on a 0-4- point scale; severity and frequency ratings are then multiplied to provide an overall score for each symptom. Minimum score is 0 (symptom is absent); maximum score is 176 (11 items × maximum severity 4 × maximum frequency 4). Higher scores indicate increased frequency or severity of behavioral issues while lower scores indicate decreased severity and frequency. | Changes from Baseline to 6 months (PBA F*S) | Posted | Mean | Standard Deviation | points on a scale | Change from Baseline in Problem Behaviors Assessment short form at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Motor Function by Using the Unified Parkinson's Disease Rating Scale (UPDRS)-I-III. | UPDRS-I-III is used to follow the longitudinal course of Parkinson's disease. The UPDRS is made up of these sections: Part I: evaluation of mentation, behavior, and mood. Part II: self-evaluation of the activities of daily life (ADLs) Part III: clinician-scored monitored motor evaluation. Part IV: complications of therapy. Part V: Hoehn and Yahr staging of severity of Parkinson's disease. The minimum possible score on the UPDRS is 0, which indicates no disability or no symptoms of Parkinson's disease. The scale, used to assess severity, typically ranges from 0 to 199 (total) (199 being the maximum) or 0 to 108 (motor section, Part III) (108 being maximum). Higher scores on the Unified Parkinson's Disease Rating Scale (UPDRS) Parts I-III indicate increased severity of disease, greater disability, and more significant impairment, while lower scores signify better function. | Changes from Baseline to 6 months | Posted | Mean | Standard Deviation | points on a scale | Change from Baseline in Unified Parkinson's Disease Rating Scale (UPDRS)-I-III at 6 months |
|
|
|
| Other Pre-specified | Measure the Effects of Nilotinib on Motor Function by Using the Timed-Up-And-Go (TUG). | Timed Up and Go (TUG) is an assessment of mobility, balance, walking ability, and fall risk. It measures the time that a person takes to rise from a chair, walk three meters, turn around, walk back to the chair, and sit down. This assessment is measured in seconds. A score of less than 10 seconds is normal, 14-20 seconds indicates a high fall risk or frailty, and over 30 seconds suggests significant mobility impairment. | Changes form Baseline to 6 months | Posted | Mean | Standard Deviation | seconds | Change from Baseline in Timed-Up-And-Go at 6 months |
|
|
|
| 0 |
| 22 |
| 2 |
| 22 |
| 15 |
| 22 |
| EG001 | 200 mg Nilotinib | Nilotinib Oral Capsule: Twenty-one (21) patients in arm 2 received the 200 mg of Nilotinib; one (1) capsule orally (without food) once daily for 6 months (180 days). | 0 | 21 | 2 | 21 | 11 | 21 |
| Appendectomy | Gastrointestinal disorders | Systematic Assessment |
|
| Dyskinesia | Nervous system disorders | Systematic Assessment | Occurred at Baseline (before study drug was administered). |
|
| Fall | Injury, poisoning and procedural complications | Systematic Assessment | Occurred at Baseline (before study drug was administered). |
|
| COVID-19 | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Ecchymosis | Cardiac disorders | Systematic Assessment |
|
| Joint and Muscle pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Hallucinations | Nervous system disorders | Systematic Assessment |
|
| Urinary Tact Infection | Renal and urinary disorders | Systematic Assessment |
|
| Tissue mass | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Lesions | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
Not provided
Not provided
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| Total alpha synuclein |
|
| Matrix metalloprotease-10 |
|
| Change from Baseline: Frontal Cortex |
|
| Baseline: Temporal Lobe |
|
| 6 months: Temporal Lobe |
|
| Change from Baseline: Temporal Lobe |
|
| End of Treatment (EOT) |
|
| MOCA (6mths) |
|
| TMT (6 mths) |
|
| ADAS-Cog (6mths) |
|
| ADCS-ADL (6mths) |
|
| NPI (6mths) |
|
| CAF Total (6mths) |
|
| IAS (6mths) |
|
| PBA F*S (6mths) |
|
| (UPDRS)-I-III (6mths) |
|
| TUG (6mths) |
|