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This study investigates the safety and tolerability of drug IkT-148009 in untreated Parkinson's disease volunteers (30 to 80 years old). It also looks at the pharmacokinetics of IkT-148009 in the body and evaluates the effect of IkT-148009 on motor and non-motor features of the disease. This 12 week study is designed to be 3:1 randomized across 3 doses of IkT-148009 or placebo. Each participant will self-administer one of 3 doses or placebo of IkT-148009 once daily (QD) with food for 12 weeks. For more information, visit our website: www.the201trial.com
This is a 12-Week, randomized, double-blind, multi-center, placebo-controlled dose-ranging clinical trial of three IkT 148009 doses in patients with untreated PD designed to assess safety, tolerability, and pharmacokinetics of IkT-148009, an oral, once daily c-Abl tyrosine kinase inhibitor. Secondary and exploratory assessments will evaluate the effect of IkT-148009 on motor and non-motor features of the disease. 120 participants are anticipated to be enrolled at up to 34 sites across the US.
Participants will undergo screening to evaluate their eligibility to participate in the study to include evaluation of Parkinson's diagnosis, vital signs, blood chemistry, hematology and urinalysis and complete listing of concomitant medications. An Enrollment Authorization Committee (EAC) will be responsible for reviewing screening data and confirming the eligibility and suitability of participants. Those selected will be enrolled and randomized to one of three active IkT-148009 arms (50/100/200 mg) or a placebo arm (1:1:1:1). All clinical staff, study investigators, and participants will be blinded to study assignments throughout the trial.
A Data Safety Monitoring Committee (DSMB) will evaluate all available safety, tolerability, and PK and Parkinson's disease-related data for each cohort on a monthly to quarterly basis. Adverse event reporting will be evaluated in real-time.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 50mg IkT-148009 (risvodetinib) | Experimental | This arm consisted of participants treated with the 50mg dose of risvodetinib. |
|
| 100mg IkT-148009 (risvodetinib) | Experimental | This arm consisted of participants treated with the 100mg dose of risvodetinib. |
|
| 200mg IkT-148009 (risvodetinib) | Experimental | This arm consisted of participants treated with the 200mg dose of risvodetinib. |
|
| Placebo | Placebo Comparator | This arm consisted of participants treated with placebo. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IkT-148009 (risvodetinib) | Drug | Oral administration gelatin capsule |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-emergent Adverse Events (TEAEs) | Baseline to 12 weeks | |
| Proportion of Those Randomized in Each Dosing Cohort Who Discontinued the Assigned Regimen Due to an Adverse Event | Baseline to 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| LS Mean of Change From Baseline in MDS-UPDRS Part II+III From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Parts II and III reflect assessments covering activites of daily living as reported by the participant (Part II) as well as a clinicians assessment of motor abilities (Part III). Part II consists of 13 items with total scores ranging from 0 to 52 while Part III consists of 18 items with total scores range from 0 to 132. Each item is scored 0 (normal) to 4 (severe). The sum of Parts 2 and 3 have a score range of 0 to 184 (31 total items). An increase in sum of Parts 2 and 3 relative to baseline represents a worsening of the participant's Parkinson's disease |
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Inclusion Criteria
Sex and Contraceptive/Barrier Requirements:
Informed Consent:
1. Capable of giving signed ICF as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
Other Inclusions:
1. Approved as an appropriate and suitable candidate by the EAC.
Exclusion Criteria
Prior/Concomitant Therapy:
Prior/Concurrent Clinical Study Experience:
Diagnostic Assessments:
Active suicidal ideation within one year prior to screening visit, as determined by the Columbia Suicide Rating Scale (answer of "yes" on question 4 or 5)
Current diagnosis or history of substance abuse (excluding nicotine or caffeine) by Diagnostic and Statistical Manual of Mental Disorders (DSM)-5 criteria
Medical or recreational use of marijuana in the 3 months prior to the screening visit
Any social or behavioral reason that would preclude completion of the study, in the judgement of the investigator
Any skin condition that would interfere with obtaining adequate samples
Evidence of advanced, age-related macular degeneration (neovascular or geographic atrophy) or intermediate macular degeneration as defined by Beckman classification (Large drusen > 125 um and/or any AMD pigmentary abnormalities). Evidence of retina/choroid neovascularization from any cause. Evidence of central serous retinopathy.
Abnormal amylase and/or lipase at screening (may be repeated during screening period)
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels greater than 2.5 times the upper limit of normal (ULN)
Significant renal impairment as determined by the following criteria:
Currently lactating, pregnant or planning on becoming pregnant during the study
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| Name | Affiliation | Role |
|---|---|---|
| Milton Werner, PhD | ABLi Therapeutics, Inc. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurology | Scottsdale | Arizona | 85258 | United States | ||
| Neurology |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41721133 | Derived | Werner MH, McGarry A, Meyer C, Mancino E, Klint C, Pellecchia J, Kieburtz K, Levine T, Bellaire B, Gibbons C, Freeman R, Ellenbogen A, Klos K, Ospina M, Hauser RA, Shannon K, Asaad H, Park A, McAllister P, Isaacson SH, LeDoux MS, Dhall R, Shpiner D, Charles P, Agarwal P, Peckham E, Mazzeo P, Davis M, Pahwa R, Brodsky M, Lew M, Purino L, Mantri S, Parashos S, Justiz W, Chachar M, Robottom B, Budman E, Blindauer K, Bellows S, Goudreau J, Steen S, Olanow CW. The 201 Trial: a placebo-controlled randomized phase 2 study of safety and tolerance of the c-Abl kinase inhibitor risvodetinib in untreated Parkinson's disease. Nat Aging. 2026 Mar;6(3):626-635. doi: 10.1038/s43587-026-01084-4. Epub 2026 Feb 20. |
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End of study
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Participants were recruited from 32 clinical sites in the United States including both private clinics and public institutions. A total of 137 participants were enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | 50mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 50mg dose of risvodetinib for 12 weeks. |
| FG001 | 100mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 100mg dose of risvodetinib for 12 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 19, 2023 | Sep 16, 2025 |
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| Placebo | Drug | Oral administration gelatin capsule |
|
| Baseline to Week 12 |
| LS Mean of Change From Baseline in PDQ-39 From a Mixed Model for Repeated Measures | Parkinson's Disease Questionnaire (PDQ) consists of 39 questions evaluating activities of daily living. Participants rate each question on a 5 point scale (0 to 4), . The PDQ-39 score ranges from 0 to 100 with higher score representing more advanced disease. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in PGI-S From a Mixed Model for Repeated Measures | Patient Global Impression - Severity (PGI-S) is participant reported assessment designed to evaluate the severity of their Parkinson's Disease. Scores range from 1 to 4 with 1 considered Normal and 4 considered Severe. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in CGI-S From a Mixed Model for Repeated Measures | Clinician Global Impression - Severity (CGI-S) is clinician reported assessment designed to evaluate the severity of a participants Parkinson's disease. Scores range from 1 to 7 with 1 considered "Normal" and 7 considered "Among the most extremely ill patients". | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in MDS-UPDRS Part II From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part II assesses activities of daily living as reported by the participant. Part II consists of 13 items with a total score ranging from 0 to 52. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | Baseline to 12 weeks |
| LS Mean of Change From Baseline in MDS-UPDRS Part III From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part III is completed by clinician and assesses a participants motor function. Part III consists of 18 items with a total score ranging from 0 to 132. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | Baseline to 12 weeks |
| LS Mean of Change From Baseline in MDS-UPDRS Part I From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part I assesses non-motor experiences of daily living. Part I is made up of two section with one rated by the investigator and another completed by the participant. Part I consists of 13 items with a total score ranging from 0 to 52. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in NMSS From a Mixed Model for Repeated Measures | Non-Motor Symptom Score (NMSS) is a 30 question assessment completed by the clinician designed to evaluate the non-motor symptoms of Parkinson's disease. Scores range from 0 to 360 with higher scores reflect increasing severity of disease | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in CSBM Score From a Mixed Model for Repeated Measures | The Complete Spontaneous Bowel Movement (CSBM) Diary is a participant-completed paper diary used to record bowel movement activity over a 7-day period. Each entry captures the date and time of the bowel movement, stool consistency (Bristol Stool Form Scale), and whether the bowel movement was spontaneous (no laxative or rescue medication within the previous 24 hours) and complete (a sensation of full evacuation). The weekly CSBM score is calculated as the total number of complete and spontaneous bowel movements recorded during the 7-day period. Rome IV criteria defines constipation as having less than 3 CSBMs per 7 day period. A decrease in CSBM score represents a decrease in bowel function. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in ESS From a Mixed Model for Repeated Measures | The Epworth Sleepiness Scale is a self-administered questionnaire that measures a participant's general level of daytime sleepiness. Participants rate their likelihood of dozing off or falling asleep in eight common situations using a 4-point scale from 0 = would never doze to 3 = high chance of dozing. The total ESS score ranges from 0 to 24, with higher scores indicating greater daytime sleepiness. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in SE-ADL From a Mixed Model for Repeated Measures | Schwab and England Activies of Daily Living (SE-ADL) is a clinician administered assessment whereby participants assess their ability to complete routine daily activities on a scale from 0 to 100. The score is reported in increments of 10 with 100 representing normal ability to complete daily activities. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in PAGI-SYM From a Mixed Model for Repeated Measures | The Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) is a participant-reported questionnaire designed to assess the severity of upper gastrointestinal symptoms over the previous two weeks. The instrument includes 20 items. Each item is rated on a 6-point Likert scale from 0 = none to 5 = very severe, with higher scores indicating greater symptom severity. A total score is calculated as the mean of non-missing item responses. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in PAC-QoL From a Mixed Model for Repeated Measures | The Patient Assessment of Constipation Quality of Life (PAC-QOL) is a participant-reported questionnaire that evaluates the impact of constipation and its treatment on quality of life over the previous two weeks. It consists of 28 items. Each item is rated on a 5-point Likert scale from 0 = not at all / very satisfied to 4 = extremely / very dissatisfied, depending on item phrasing. Scores are calculated as the mean of all non-missing items, with higher scores indicating greater negative impact on quality of life. | Change from Baseline to Week 12 |
| LS Mean of Change From Baseline in PAGI-QoL From a Mixed Model for Repeated Measures | The Patient Assessment of Upper Gastrointestinal Disorders Quality of Life (PAGI-QOL) is a validated, participant-reported questionnaire that assesses the impact of upper gastrointestinal symptoms on quality of life over the previous two weeks. The instrument includes 30 items. Each item is rated on a 6-point Likert scale from 0 = none of the time to 5 = all of the time. Scores are calculated as the mean of all non-missing items, with lower scores represent a greater negative impact on a participant's quality of life. | Change from Baseline to Week 12 |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Neurology | Reseda | California | 91335 | United States |
| Neurology | Stamford | Connecticut | 06905 | United States |
| Neurologist | Boca Raton | Florida | 33486 | United States |
| Neurology | Miami | Florida | 33136 | United States |
| Neurology | Naples | Florida | 34105 | United States |
| Neurology | Tampa | Florida | 33609 | United States |
| Neurology | Tampa | Florida | 33613 | United States |
| Neurology | Foxborough | Massachusetts | 02035 | United States |
| Neurology | South Dartmouth | Massachusetts | 02747 | United States |
| Neurology | Farmington Hills | Michigan | 48334 | United States |
| Neurology | Golden Valley | Minnesota | 55427 | United States |
| Neurology | West Long Branch | New Jersey | 07764 | United States |
| Neurology | Durham | North Carolina | 27705 | United States |
| Neurology | Raleigh | North Carolina | 27607 | United States |
| Neurology | Columbus | Ohio | 43221 | United States |
| Neurology | Tulsa | Oklahoma | 74136 | United States |
| Neurology | Portland | Oregon | 97239 | United States |
| Neurology | Port Royal | South Carolina | 29935 | United States |
| Neurology | Memphis | Tennessee | 38137 | United States |
| Neurology | Nashville | Tennessee | 37232 | United States |
| Neurology | Frisco | Texas | 75035 | United States |
| Neurology | Houston | Texas | 77030 | United States |
| Neurology | Round Rock | Texas | 78681 | United States |
| Neurology | Kirkland | Washington | 98034 | United States |
| Neurology | Madison | Wisconsin | 53705 | United States |
| Neurology | Milwaukee | Wisconsin | 53226 | United States |
| FG002 | 200mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks. |
| FG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 50mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 50mg dose of risvodetinib for 12 weeks. |
| BG001 | 100mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 100mg dose of risvodetinib for 12 weeks. |
| BG002 | 200mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks. |
| BG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Hoehn & Yahr | Clinician's assessment of a particiapnt's disease severity at the time of screening. The scale is based on a rating of 0-5 with 0 representing no signs of disease and 5 representing a participant that is wheelchair bound or bedridden. | Count of Participants | Participants | No |
|
| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | LS Mean of Change From Baseline in MDS-UPDRS Part II+III From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Parts II and III reflect assessments covering activites of daily living as reported by the participant (Part II) as well as a clinicians assessment of motor abilities (Part III). Part II consists of 13 items with total scores ranging from 0 to 52 while Part III consists of 18 items with total scores range from 0 to 132. Each item is scored 0 (normal) to 4 (severe). The sum of Parts 2 and 3 have a score range of 0 to 184 (31 total items). An increase in sum of Parts 2 and 3 relative to baseline represents a worsening of the participant's Parkinson's disease | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Score on the Scale | Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in PDQ-39 From a Mixed Model for Repeated Measures | Parkinson's Disease Questionnaire (PDQ) consists of 39 questions evaluating activities of daily living. Participants rate each question on a 5 point scale (0 to 4), . The PDQ-39 score ranges from 0 to 100 with higher score representing more advanced disease. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment Score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in PGI-S From a Mixed Model for Repeated Measures | Patient Global Impression - Severity (PGI-S) is participant reported assessment designed to evaluate the severity of their Parkinson's Disease. Scores range from 1 to 4 with 1 considered Normal and 4 considered Severe. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in CGI-S From a Mixed Model for Repeated Measures | Clinician Global Impression - Severity (CGI-S) is clinician reported assessment designed to evaluate the severity of a participants Parkinson's disease. Scores range from 1 to 7 with 1 considered "Normal" and 7 considered "Among the most extremely ill patients". | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in MDS-UPDRS Part II From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part II assesses activities of daily living as reported by the participant. Part II consists of 13 items with a total score ranging from 0 to 52. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Scale score | Baseline to 12 weeks |
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| Secondary | LS Mean of Change From Baseline in MDS-UPDRS Part III From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part III is completed by clinician and assesses a participants motor function. Part III consists of 18 items with a total score ranging from 0 to 132. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment Score | Baseline to 12 weeks |
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| Secondary | LS Mean of Change From Baseline in MDS-UPDRS Part I From a Mixed Model for Repeated Measures | Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS). Part I assesses non-motor experiences of daily living. Part I is made up of two section with one rated by the investigator and another completed by the participant. Part I consists of 13 items with a total score ranging from 0 to 52. Each item is scored 0 (normal) to 4 (severe). Higher scores reflect increased severity of disease. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assesment Score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in NMSS From a Mixed Model for Repeated Measures | Non-Motor Symptom Score (NMSS) is a 30 question assessment completed by the clinician designed to evaluate the non-motor symptoms of Parkinson's disease. Scores range from 0 to 360 with higher scores reflect increasing severity of disease | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in CSBM Score From a Mixed Model for Repeated Measures | The Complete Spontaneous Bowel Movement (CSBM) Diary is a participant-completed paper diary used to record bowel movement activity over a 7-day period. Each entry captures the date and time of the bowel movement, stool consistency (Bristol Stool Form Scale), and whether the bowel movement was spontaneous (no laxative or rescue medication within the previous 24 hours) and complete (a sensation of full evacuation). The weekly CSBM score is calculated as the total number of complete and spontaneous bowel movements recorded during the 7-day period. Rome IV criteria defines constipation as having less than 3 CSBMs per 7 day period. A decrease in CSBM score represents a decrease in bowel function. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Number of bowel movements per week | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in ESS From a Mixed Model for Repeated Measures | The Epworth Sleepiness Scale is a self-administered questionnaire that measures a participant's general level of daytime sleepiness. Participants rate their likelihood of dozing off or falling asleep in eight common situations using a 4-point scale from 0 = would never doze to 3 = high chance of dozing. The total ESS score ranges from 0 to 24, with higher scores indicating greater daytime sleepiness. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment Score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in SE-ADL From a Mixed Model for Repeated Measures | Schwab and England Activies of Daily Living (SE-ADL) is a clinician administered assessment whereby participants assess their ability to complete routine daily activities on a scale from 0 to 100. The score is reported in increments of 10 with 100 representing normal ability to complete daily activities. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment Score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in PAGI-SYM From a Mixed Model for Repeated Measures | The Patient Assessment of Upper Gastrointestinal Disorders-Symptom Severity Index (PAGI-SYM) is a participant-reported questionnaire designed to assess the severity of upper gastrointestinal symptoms over the previous two weeks. The instrument includes 20 items. Each item is rated on a 6-point Likert scale from 0 = none to 5 = very severe, with higher scores indicating greater symptom severity. A total score is calculated as the mean of non-missing item responses. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in PAC-QoL From a Mixed Model for Repeated Measures | The Patient Assessment of Constipation Quality of Life (PAC-QOL) is a participant-reported questionnaire that evaluates the impact of constipation and its treatment on quality of life over the previous two weeks. It consists of 28 items. Each item is rated on a 5-point Likert scale from 0 = not at all / very satisfied to 4 = extremely / very dissatisfied, depending on item phrasing. Scores are calculated as the mean of all non-missing items, with higher scores indicating greater negative impact on quality of life. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment Score | Change from Baseline to Week 12 |
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| Secondary | LS Mean of Change From Baseline in PAGI-QoL From a Mixed Model for Repeated Measures | The Patient Assessment of Upper Gastrointestinal Disorders Quality of Life (PAGI-QOL) is a validated, participant-reported questionnaire that assesses the impact of upper gastrointestinal symptoms on quality of life over the previous two weeks. The instrument includes 30 items. Each item is rated on a 6-point Likert scale from 0 = none of the time to 5 = all of the time. Scores are calculated as the mean of all non-missing items, with lower scores represent a greater negative impact on a participant's quality of life. | The mITT population will include all randomized participants who have a valid baseline MDS-UPDRS, received at least one dose of study drug, and have at least one post-baseline evaluation of the MDS-UPDRS assessment as defined in the Statistical Analysis Plan | Posted | Least Squares Mean | 95% Confidence Interval | Assessment score | Change from Baseline to Week 12 |
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| Primary | Incidence of Treatment-emergent Adverse Events (TEAEs) | Posted | Count of Participants | Participants | Baseline to 12 weeks |
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| Primary | Proportion of Those Randomized in Each Dosing Cohort Who Discontinued the Assigned Regimen Due to an Adverse Event | Posted | Count of Participants | Participants | Baseline to 12 weeks |
|
|
Enrollment until the end of follow up (14 weeks post baseline visit)
All adverse event recordings were done in compliance with ICH and FDA guidance
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 50mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 50mg dose of risvodetinib for 12 weeks. | 0 | 35 | 1 | 35 | 19 | 35 |
| EG001 | 100mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 100mg dose of risvodetinib for 12 weeks. | 0 | 34 | 0 | 34 | 21 | 34 |
| EG002 | 200mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks. | 0 | 33 | 1 | 33 | 21 | 33 |
| EG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. | 0 | 35 | 1 | 35 | 21 | 35 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Femur Fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment | Not related |
|
| Infection of Finger | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment | Not Related |
|
| Diverticulitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment | Not Related |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Blood Creatinine Increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Lipase Increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Neck Pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Orthostatic Hypotension | Vascular disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Atrial Flutter | Cardiac disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Ventricular extrasystoles | Cardiac disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Diplopia | Eye disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Eye Swelling | Eye disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Ocular Discomfort | Eye disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Photopsia | Eye disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Abdominal pain lower | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Abdominal Pain upper | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Anal Incontinence | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Gastroesophageal reflux disease | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Pneumoperitoneum | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Adverse drug reaction | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Chest Discomfort | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Malaise | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Pain | General disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Bacteriuria | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Diverticulitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Ear Infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Localized Infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Postoperative wound infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Subcutaneous Abscess | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Arthopod bite | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dental restoration failure | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Eye contusion | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Femur Fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Post procedural erythema | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 21.0 | Non-systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Blood oestrogen increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Blood urea increased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Glomerular filtration rate decreased | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Glucose urine present | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Urine leukocyte esterase | Investigations | MedDRA 21.0 | Non-systematic Assessment |
| |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Muscle twitching | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Tenosynovitis | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Seborrhoeic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.0 | Non-systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 21.0 | Non-systematic Assessment |
| |
| Action tremor | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Carpal tunnel syndrome | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Intention tremor | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Lumbar radiculopathy | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Restless leg syndrome | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Typical aura without headache | Nervous system disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Disturbance in social behavior | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Somnmbulism | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Stress | Psychiatric disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Hypertonic bladder | Renal and urinary disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Obstructive sleep apnea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Actinic keratosis | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Skin discoloration | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 21.0 | Non-systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA 21.0 | Non-systematic Assessment |
|
Upon completion of the Study and evaluation by Sponsor of all data from the Study, or upon early termination or abandonment of the Study, Institution and Investigator may publish or otherwise publicly disclose, for non-commercial purposes, Study Data .
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Milton Werner | ABLi Therapeutics | 9174940831 | info@ablitherapeutics.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 28, 2025 | Sep 16, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
Not provided
Not provided
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| H&Y 2.0 |
|
| H&Y 1.5 |
|
| H&Y 1.0 |
|
| Missing |
|
Change relative to placebo from baseline to 12 weeks of treatment
| Mixed Models Analysis |
| 0.606 |
Study not sized to evaluate efficacy |
| Mean Difference (Net) |
| 0.97 |
| Standard Error of the Mean |
| 1.876 |
| 2-Sided |
| 95 |
| -2.745 |
| 4.687 |
| Superiority |
| Change relative to placebo from baseline to 12 weeks of treatment | Mixed Models Analysis | 0.509 | Study not sized to evaluate efficacy | Mean Difference (Net) | 1.27 | Standard Error of the Mean | 1.925 | 2-Sided | 95 | -2.539 | 5.086 | Superiority |
| OG003 |
| Placebo |
This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| Placebo |
This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 |
| Placebo |
This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| Placebo |
This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG002 | 200mg IkT-148009 (Risvodetinib) | This arm consisted of participants treated with the 200mg dose of risvodetinib. |
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks.
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks. |
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
This arm consisted of participants treated with the 200mg dose of risvodetinib for 12 weeks. |
| OG003 | Placebo | This arm will consist participants treated with placebo for 12 weeks. |
|
|
|
|
|
|