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Interim analysis was performed for 442 patients who completed Part I of the PROSEEK study-(CLR_18_06); it did not show evidence of treatment benefit in patients receiving Vodobatinib.
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| Name | Class |
|---|---|
| Sun Pharma Advanced Research Company Limited | INDUSTRY |
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This study evaluates the safety and tolerability of treatment with K0706 in Dementia with Lewy Bodies (DLB).
The hypothesis is that K0706 will be safe and tolerable and that this drug will alter CSF and plasma biomarkers in DLB. Clinical assessments of cognitive, behavioral and motor functioning will also be evaluated. A total of 45 participants will be randomized 1:1:1 into 3 groups (n=15/per group) to be treated with sachet of 192 mg powder of K0706 ( equivalent to 96 mg capsule of K0706) or sachet of 384 mg powder of K0706 (equivalent to 192 capsule of K0706) or sachet of matching placebo ( equivalent to a capsule of placebo) for 12 weeks, followed by 4-week wash-out period.
Dementia with Lewy Bodies (DLB) is an alpha-synucleinopathy 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). DLB and PD are characterized by death of dopaminergic (DA) neurons in the nigro-striatal system and formation of intra-neuronal alpha-synuclein inclusions known as Lewy bodies (LBs). Misfolded alpha-synuclein aggregates within LBs and apha-synuclein (SYN) is the highest genetic risk factor for PD and DLB followed by the microtubule associated protein tau (MAPT) . At autopsy alpha-synuclein, hyper-phosphorylated tau (p-tau) and amyloid plaques are all detected in the brains of individuals with DLB. Therefore, the neuropathology of DLB overlaps with both PD and AD, and includes alpha-synuclein accumulation in LBs, p-tau and beta-amyloid deposition . Potential cerebrospinal fluid (CSF) biomarkers, including alpha-synuclein, dopamine metabolites homovanillic acid (HVA) and 3,4-Dihydroxyphenylacetic acid (DOPAC) , total tau and p-tau and amyloid beta peptides (Abeta 40/42) may be commonly shared in AD, PD and DLB. The core clinical features of DLB, include dementia and Parkinsonism in addition to hallucinations, cognitive fluctuations and rapid eye movement (REM) sleep behavior disorders (RBD) . L-Dopa replacement therapies and acetylcholinesterase inhibitors may partially control motor and cognitive symptoms, respectively in DLB. Selective Serotonin Re-uptake Inhibitors (SSRIs) and antipsychotics manage the behavioral but worsen motor symptoms in DLB. There is a major unmet medical need for further research into DLB to identify potential therapies for this disease and provide significant insights into the treatment of other Parkinsonian and memory disorders. A major challenge facing DLB is to develop a therapy that can halt neuronal death and alleviate cognitive, motor and behavioral symptoms. No therapeutic approach exists to alter the levels of neurotoxic proteins such as alpha-synuclein and halt DA and other neuronal death in DLB. One mechanism to degrade neurotoxic proteins is autophagy , which is a process by which the cell can degrade its own contents. There is evidence that autophagy is impaired in neurodegeneration , leading to failure of degradation of protein aggregates, including misfolded alpha-synuclein. Importantly, autophagy is exploited therapeutically in several diseases, including adult chronic myeloid leukemia (CML). Tyrosine kinase inhibitors (TKIs) induce autophagy, leading to destruction of rapidly dividing tumor cells in CML and degradation of neurotoxic proteins, including alpha-synuclein, beta-amyloid and p-tau in PD and AD models. Sun Pharma Advanced Research Company Limited (SPARC Ltd.) is developing K0706, for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) that is resistant or intolerant to prior TKI therapy or Philadelphia positive acute lymphoblastic leukemia (Ph+ ALL) that is resistant or intolerant to prior TKI therapy; and its ability to slow down progression of PD. Using allometric scaling and an average human body weight of 70kg an oral dose of 15-30mg/kg once daily in mice corresponds to an oral human equivalent dose (HED) of 85-160 mg which is within the tolerated dose in both CML and PD. Therefore, the effects of a sachet of 192 mg powder of K0706 (equivalent to 96 mg capsule of K0706 ) and a sachet of 384 mg powder of K0706( equivalet to 192 mg capsule of K0706) and a sachet of matching placebo taken daily by mouth for 12 weeks, followed by a 4 week wash-out period will be evaluated in individuals diagnosed with DLB. The data obtained from this study will serve as a proof of concept for future placebo-controlled, double-blind studies in patients diagnosed with DLB, AD, or PD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo powder | Placebo Comparator | Forty five (45) participants will be recruited and randomized into 3 arms (1:1:1). Fifteen (15) patients in arm 1 (group 1) will receive the matching placebo powder orally once daily for 12 weeks (90 days). |
|
| 192 mg powder of K0706 | Active Comparator | Forty five (45) participants will be recruited and randomized into 3 arms (1:1:1) .Fifteen (15) patients in arm 2 (group 2) will receive the 192 mg powder of K0706 ( equivalent to 96 mg capsule of K0706) orally once daily for 12 weeks (90 days). |
|
| 384 mg powder of K0706 | Active Comparator | Forty five (45) participants will be recruited and randomized into 3 arms (1:1:1) .Fifteen (15) patients in arm 3 (group 3) will receive the 384 mg powder of K0706 (equivalent to 192 mg capsule of K0706) orally once daily for 12 weeks(90 days). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Fifteen (15) patients in group 1 will receive the sachet of matching placebo ( equivalent to a capsule of placebo"sugar pill") orally daily for 12 weeks (90 days) without food. |
| Measure | Description | Time Frame |
|---|---|---|
| Evidence of Treatment-emergent Adverse Effects (Safety and Tolerability) | The investigators will determine safety and tolerability by using the occurrence of adverse events (AEs) of interest, including myelosuppression, urinary, pancreatic and hepatic disorders, gastrointestinal (GI), kidney disorders, Corrected QT-interval (QTc)prolongation as per SPARC Ltd Investigator Brochure (IB). | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Measurement of Plasma Biomarkers in DLB Patients | DLB related plasma biomarkers, including alpha-synuclein, HVA, DOPAC were measured at Baseline and 12 weeks. | Baseline, End of Treatment (EOT), and the change between baseline and EOT |
| Measurement of Biomarker Concentration in CSF |
| Measure | Description | Time Frame |
|---|---|---|
| Measurement of the Effects of K0706 on Cognition Using the Montreal Cognitive Assessment (MoCA) at Baseline and 12 Weeks | 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 where 30 is the highest score and 0 is the lowest score. |
Inclusion Criteria:
Exclusion Criteria:
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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 |
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| Label | URL |
|---|---|
| https://sites.google.com/a/georgetown.edu/moussa-lab/home3 | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo Powder | Placebo: Ten (10) patients in group 1 received the sachet of matching placebo (equivalent to a capsule of placebo "sugar pill") orally daily for 12 weeks (90 days) without food. |
| FG001 | 192 mg Powder of K0706 | 192 mg powder of K0706: Ten (10) patients in group 2 received the sachet of 192 mg powder of K0706 (equivalent to 96 mg capsule of K0706) orally for 12 weeks (90 days) without food. |
| FG002 | 384 mg Powder of K0706 | 384 mg powder of K0706: Nine (9) patients in group 3 received the 384 mg powder of K0706 (equivalent to 192 capsule of K0706) orally daily for 12 weeks (90 days) without food. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo Powder | Placebo: Ten (10) patients in group 1 received the sachet of matching placebo (equivalent to a capsule of placebo "sugar pill") orally daily for 12 weeks (90 days) without food. |
| BG001 | 192 mg Powder of K0706 |
| 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 | Evidence of Treatment-emergent Adverse Effects (Safety and Tolerability) | The investigators will determine safety and tolerability by using the occurrence of adverse events (AEs) of interest, including myelosuppression, urinary, pancreatic and hepatic disorders, gastrointestinal (GI), kidney disorders, Corrected QT-interval (QTc)prolongation as per SPARC Ltd Investigator Brochure (IB). | Posted | Number | events | 12 weeks |
|
Adverse events were recorded over the course of sixteen weeks for each participant.
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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 Powder | Placebo: Ten (10) patients in group 1 received the sachet of matching placebo (equivalent to a capsule of placebo "sugar pill") orally daily for 12 weeks (90 days) without food. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| congestive heart failure and aspiration pneumonia | Cardiac disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Falls | General disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Fernando Pagan | Georgetown University | 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 | Dec 6, 2022 | Sep 9, 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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The effects of 192 mg powder of K0706 ( equivalent to 96 mg capsule of K0706) versus 384 mg powder of K0706 (equivalent to 192 mg capsule of K0706) versus matching placebo powder taken daily by mouth for 12 weeks, followed by a 4 week wash-out period in individuals diagnosed with DLB will be evaluated.
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Participants will be randomized by a Biostatistician by an internet based randomization module into three groups. The researchers include : Primary investigator , Sub-Investigators ,Clinical Coordinators, Nurse Practitioners, and Clinical Research unit (CRU) staff.
The investigators will be blinded to the dosage. Medications for any patient will be labeled by the CRU with a package medical identification number (Med. Id). A patient specific patient identification number (Pat. Id.) will be assigned to each patient. The investigator will have to note the Pat.Id on the designated medication package number after randomization.
| 192 mg powder of K0706 | Drug | Fifteen (15) patients in group 2 will receive the sachet of 192 mg powder of K0706 ( equivalent to 96 mg capsule of K0706) orally for 12 weeks (90 days) without food. |
|
| 384 mg powder of K0706 | Drug | Fifteen (15) patients in group 3 will receive the 384 mg powder of K0706 (equivalent to 192 capsule of K0706) orally daily for 12 weeks (90 days) without food. |
|
Concentration of DLB related CSF biomarkers, including HVA, DOPAC, Abeta40/42, total tau, ptau231/181 and total and oligomeric alpha-synuclein were measured at Baseline and 12 weeks. |
| Baseline, End of Treatment (EOT) (12 weeks), and the change between baseline and EOT |
| Measurement of Plasma Biomarkers in DLB Patients | Ratio of HVA to Dopamine, and Dopamine to HVA in the plasma at Baseline, and 12 weeks The ratio of HVA to DA is a biochemical marker used to measure dopamine turnover, reflecting the rate at which dopamine is synthesized, released, and subsequently broken down in the central nervous system. High Ratio: Indicates rapid metabolism/degradation of dopamine. Low Ratio: May suggest lower turnover or reduced activity in dopaminergic pathways. HVA/DOPAC Ratio (Optimal): 0.1 - 1.8.; a ratio above 1.8 suggests accelerated turnover, typically seen in Parkinson's Disease | Baseline, End of Treatment (EOT), and the change between baseline and EOT |
| Measurement of CSF Biomarkers in DLB Patients | Ratio of DLB related CSF biomarkers, including Abeta42 to Abeta40, phospho-Tau(181) to Abeta42, and phospho-tau181 to Total Tau at Baseline and 12 weeks. Ab42/Ab40 ratio is a biomarker used to assess Alzheimer's disease (AD) risk. A lower ratio indicates a higher likelihood of amyloid plaque accumulation in the brain and AD pathology. The ptau(181)/Ab42 ratio is a clinical biomarker used to identify AD pathology by measuring the balance between tau protein phosphorylation and amyloid-beta accumulation. Higher ratio values typically indicate a higher likelihood of amyloid and tau pathology in the brain. P-tau181 and its ratio to total tau (t-tau) or other amyloid markers are effective indicators of AD pathology. Reference Range/Ratio: A 95% reference interval for the p-tau181/t-tau ratio has been reported as 0.38-6.34, with higher values indicating a higher likelihood of AD pathology. | Baseline, End of Treatment (EOT) (12 weeks), and the change between baseline and EOT |
| Baseline, 12 weeks (EOT), and change between Baseline and End of Treatment (EOT) |
| Measurement of the Effects of K0706 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. | 12 weeks |
| 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. | 12 weeks |
| Measuring the Effects of K0706 on Behavior Using the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale. | 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; 0 is the minimum score and 78 is the maximum score. | 12 weeks |
| Measuring the Effects of K0706 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. | 12 weeks |
| Measuring the Effects of K0706 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. | 12 weeks |
192 mg powder of K0706: Ten (10) patients in group 2 received the sachet of 192 mg powder of K0706 (equivalent to 96 mg capsule of K0706) orally for 12 weeks (90 days) without food.
| BG002 | 384 mg Powder of K0706 | 384 mg powder of K0706: Nine (9) patients in group 3 received the 384 mg powder of K0706 (equivalent to 192 capsule of K0706) orally daily for 12 weeks (90 days) without food. |
| BG003 | 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 |
|
| Region of Enrollment | Number | participants |
|
| OG002 | 384 mg Powder of K0706 | 384 mg powder of K0706: Nine (9) patients in group 3 received the 384 mg powder of K0706 (equivalent to 192 capsule of K0706) orally daily for 12 weeks (90 days) without food. |
|
|
| Secondary | Measurement of Plasma Biomarkers in DLB Patients | DLB related plasma biomarkers, including alpha-synuclein, HVA, DOPAC were measured at Baseline and 12 weeks. | The mean difference within groups at baseline and end of treatment. | Posted | Mean | Standard Deviation | pg/ml | Baseline, End of Treatment (EOT), and the change between baseline and EOT |
|
|
|
| Secondary | Measurement of Biomarker Concentration in CSF | Concentration of DLB related CSF biomarkers, including HVA, DOPAC, Abeta40/42, total tau, ptau231/181 and total and oligomeric alpha-synuclein were measured at Baseline and 12 weeks. | The mean difference within groups at baseline and end of treatment. | Posted | Mean | Standard Deviation | pg/ml | Baseline, End of Treatment (EOT) (12 weeks), and the change between baseline and EOT |
|
|
|
| Secondary | Measurement of Plasma Biomarkers in DLB Patients | Ratio of HVA to Dopamine, and Dopamine to HVA in the plasma at Baseline, and 12 weeks The ratio of HVA to DA is a biochemical marker used to measure dopamine turnover, reflecting the rate at which dopamine is synthesized, released, and subsequently broken down in the central nervous system. High Ratio: Indicates rapid metabolism/degradation of dopamine. Low Ratio: May suggest lower turnover or reduced activity in dopaminergic pathways. HVA/DOPAC Ratio (Optimal): 0.1 - 1.8.; a ratio above 1.8 suggests accelerated turnover, typically seen in Parkinson's Disease | The mean difference within groups at baseline and end of treatment. | Posted | Mean | Standard Deviation | ratio | Baseline, End of Treatment (EOT), and the change between baseline and EOT |
|
|
|
| Secondary | Measurement of CSF Biomarkers in DLB Patients | Ratio of DLB related CSF biomarkers, including Abeta42 to Abeta40, phospho-Tau(181) to Abeta42, and phospho-tau181 to Total Tau at Baseline and 12 weeks. Ab42/Ab40 ratio is a biomarker used to assess Alzheimer's disease (AD) risk. A lower ratio indicates a higher likelihood of amyloid plaque accumulation in the brain and AD pathology. The ptau(181)/Ab42 ratio is a clinical biomarker used to identify AD pathology by measuring the balance between tau protein phosphorylation and amyloid-beta accumulation. Higher ratio values typically indicate a higher likelihood of amyloid and tau pathology in the brain. P-tau181 and its ratio to total tau (t-tau) or other amyloid markers are effective indicators of AD pathology. Reference Range/Ratio: A 95% reference interval for the p-tau181/t-tau ratio has been reported as 0.38-6.34, with higher values indicating a higher likelihood of AD pathology. | The mean difference within groups at baseline and end of treatment. | Posted | Mean | Standard Deviation | ratio | Baseline, End of Treatment (EOT) (12 weeks), and the change between baseline and EOT |
|
|
|
| Other Pre-specified | Measurement of the Effects of K0706 on Cognition Using the Montreal Cognitive Assessment (MoCA) at Baseline and 12 Weeks | 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 where 30 is the highest score and 0 is the lowest score. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | score on a scale | Baseline, 12 weeks (EOT), and change between Baseline and End of Treatment (EOT) |
|
|
|
| Other Pre-specified | Measurement of the Effects of K0706 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. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | seconds | 12 weeks |
|
|
|
| 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. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | score on a scale | 12 weeks |
|
|
|
| Other Pre-specified | Measuring the Effects of K0706 on Behavior Using the Alzheimer's Disease Cooperative Study-Activity of Daily Living Scale. | 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; 0 is the minimum score and 78 is the maximum score. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | score on a scale | 12 weeks |
|
|
|
| Other Pre-specified | Measuring the Effects of K0706 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. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | score on a scale | 12 weeks |
|
|
|
| Other Pre-specified | Measuring the Effects of K0706 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. | Median and Interquartile Range (IQR) are robust, non-parametric statistics used to describe central tendency and dispersion when outliers are present. The median represents the 50th percentile, while the IQR measures the spread of the middle 50% of the data. The number shown is the median value, not the mean value. | Posted | Median | Inter-Quartile Range | seconds | 12 weeks |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 6 |
| 10 |
| EG001 | 192 mg Powder of K0706 | 192 mg powder of K0706: Ten (10) patients in group 2 received the sachet of 192 mg powder of K0706 (equivalent to 96 mg capsule of K0706) orally for 12 weeks (90 days) without food. | 0 | 10 | 0 | 10 | 6 | 10 |
| EG002 | 384 mg Powder of K0706 | 384 mg powder of K0706: Nine (9) patients in group 3 received the 384 mg powder of K0706 (equivalent to 192 capsule of K0706) orally daily for 12 weeks (90 days) without food. | 0 | 9 | 1 | 9 | 3 | 9 |
| Elevated lipase | Blood and lymphatic system disorders | Systematic Assessment |
|
| Pain | General disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | Systematic Assessment |
|
| Nausea/Vomiting | General disorders | Systematic Assessment |
|
| Elevated amylase | Blood and lymphatic system disorders | Systematic Assessment |
|
| Hypophosphatemia | Blood and lymphatic system disorders | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
|
| Leukopenia | Blood and lymphatic system disorders | Systematic Assessment |
|
| COVID-19 | General disorders | Systematic Assessment |
|
| Changes in Gait | General disorders | Systematic Assessment |
|
| Cataract | Eye disorders | Systematic Assessment |
|
| Swelling | General disorders | Systematic Assessment |
|
| Hallucinations | Psychiatric disorders | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | Systematic Assessment |
|
| Urinary tract infection | Renal and urinary disorders | Systematic Assessment |
|
| Lesions | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Fracture | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Alertness | Nervous system disorders | Systematic Assessment |
|
| Insomnia | Nervous system disorders | Systematic Assessment |
|
| Confusion | Nervous system disorders | Systematic Assessment |
|
| Lethargy | Nervous system disorders | Systematic Assessment |
|
| Sciatica | Nervous system disorders | Systematic Assessment |
|
| Paranoia | Psychiatric disorders | Systematic Assessment |
|
| Upper respiratory infections | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Episodic cardiopulmonary exertion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Skin tags | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Cellulitis | 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 |
|
| Dopamine |
|
| alpha-synuclein (Baseline) |
|
| alpha-synuclein (EOT) |
|
| Homovanillic-acid (HVA) (Baseline) |
|
| Homovanillic-acid (HVA) (EOT) |
|
| Dopamine (Baseline) |
|
| Dopamine (EOT) |
|
|
| beta-amyloid(42) (AB42) |
|
| Total (t) Tau |
|
| Phospho(p)-Tau(181) |
|
| alpha-synuclein (Baseline) |
|
| alpha-synuclein (EOT) |
|
| beta-amyloid(40) (AB40) (Baseline) |
|
| beta-amyloid(40) (AB40) (EOT) |
|
| beta-amyloid(42) (AB42) (Baseline) |
|
| beta-amyloid(42) (AB42) (EOT) |
|
| Total (t) Tau (Baseline) |
|
| Total (t) Tau (EOT) |
|
| Phospho(p)-Tau(181) (Baseline) |
|
| Phospho(p)-Tau(181) (EOT) |
|
|
| HVA/Dopamine (EOT) |
|
|
| pTau(181)/tTau |
|
| AB42/AB40 (Baseline) |
|
| AB42/AB40 (EOT) |
|
| p-Tau(181)/AB42 (Baseline) |
|
| p-Tau(181)/AB42 (EOT) |
|
| pTau(181)/tTau (Baseline) |
|
| pTau(181)/tTau (EOT) |
|
|
| MOCA(EOT) |
|
|
| TMT (EOT) |
|
|
| ADAS-cog (EOT) |
|
|
| ADCS-ADL (EOT) |
|
|
| UPDRS-I-III (EOT) |
|
|
| TUG (EOT) |
|