An Early Phase 2 Clinical Study of KSP-0243

Study Overview
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Brief Summary
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A phase 2a, randomized, double-blind, placebo-controlled, multicenter, parallel-group study to administer KSP-0243 or a placebo once daily after breakfast for 8 weeks in 100 patients with mild to moderate active ulcerative colitis.

Official Title
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An Early Phase 2 Clinical Study of KSP-0243 in Patients With Mild to Moderate Active Ulcerative Colitis

Conditions
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Colitis, Ulcerative

Intervention / Treatment
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* Drug: KSP-0243
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients aged 18 to 74 years old (both inclusive) Patients who have been given the diagnosis of ulcerative colitis for at least 12 weeks Patients with an endoscopic view typically seen with ulcerative colitis spreading > 15 cm from the anal verge Patients with mild to moderate active ulcerative colitis who meet the certain conditions Patients who have an inadequate response to a fixed-dose oral 5-ASA formulation (mesalazine or salazosulfapyridine) continued from at least 2 weeks prior to the start of the screening period Exclusion Criteria: Patients who underwent an enterectomy or are expected to require an enterectomy during the study period (except appendectomy) Patients who have or suspected to have bacterium- or parasite-induced infectious enteritis (e.g., infection with Clostridium difficile) Patients with any of the following concomitant illnesses with the severity considered inappropriate as a study patient by the principal investigator or the subinvestigator or medical history thereof: Hepatic or renal disorders or cardiovascular, endocrine, metabolic, pulmonary, gastrointestinal, neurological, urological, genitourinary, and immune diseases,

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 74 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: KSP-0243<br>Under double-blinding, KSP 0243 tablets will be orally administered. | Drug: KSP-0243<br>* Oral administration<br>|
| Placebo Comparator: Placebo<br>Under double-blinding, placebo tablets will be orally administered. | Drug: Placebo<br>* Oral administration<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical response rate based on the modified Mayo score at Week 8 | The percentage of patients who satisfied both of the following requirements: Decreases in the modified Mayo score by ≥ 30% and ≥ 2 points from baseline The rectal bleeding subscore based on the Mayo score decreases by ≥ 1 point from baseline or the subscore becomes ≤ 1 point | Up to 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical remission rate based on the modified Mayo score at Week 8 | Clinical remission rate based on the modified Mayo score: The percentage of patients who satisfied all of the following requirements: The stool frequency subscore based on the Mayo score is 0 or 1 point with no aggravation from baseline The rectal bleeding subscore based on the Mayo score is 0 points The endoscopy subscore of the Mayo score is 0 or 1 point | Up to 8 weeks | 
| Clinical response rate based on the full Mayo score at Week 8 | Clinical response rate based on the full Mayo score: The percentage of patients who satisfied all of the following requirements. Decreases in the full Mayo score by ≥ 30% and ≥ 3 points from baseline The rectal bleeding subscore based on the Mayo score decreases by ≥ 1 point from baseline or the subscore becomes ≤ 1 point | Up to 8 weeks | 
| Clinical remission rate based on the full Mayo score at Week 8 | Clinical remission rate based on the full Mayo score: The percentage of patients who satisfied all of the following requirements. Full Mayo score is ≤ 2 points All the subscores are ≤ 1 point | Up to 8 weeks | 
| Incidence of adverse events (AE) and adverse drug reactions (ADR) | - Adverse events, Adverse drug reactions | Up to 8 weeks | 
| Laboratory tests (Hematology): Hemoglobin (g/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory tests (Hematology): Hematocrit (%) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory tests (Hematology): Erythrocyte (10^10/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Leukocyte (10^6/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Neutrophil (10^8/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Eosinophil (10^8/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Basophil (10^8/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Monocyte (10^8/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Lymphocyte (10^8/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Hematology): Platelet (10^10/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Na (mEq/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): K (mEq/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Cl (mEq/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Ca (mEq/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): P (mEq/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Creatinine (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Total Bilirubin (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Direct Bilirubin (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Indirect Bilirubin (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Total Protein (g/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Albumin (g/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): AST (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): ALT (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): γGTP (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): ALP (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Creatinine kinase (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Uric acid (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): BUN (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): LDH (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Total Cholesterol (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Triglyceride (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Amylase (U/L) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Blood chemistry): Glucose (mg/dL) | Summary statistics, scattered plots before and after the dose will be presented. Shift tables before and after dose will be presented. | Up to 8 weeks | 
| Laboratory test (Urinalysis): Protein | Number and percentage of subjects will be presented. Shift tables before and after the dose will be presented. | Up to 8 weeks | 
| Laboratory test (Urinalysis): Glucose | Number and percentage of subjects will be presented. Shift tables before and after the dose will be presented. | Up to 8 weeks | 
| Laboratory test (Urinalysis): Urobilinogen | Number and percentage of subjects will be presented. Shift tables before and after the dose will be presented. | Up to 8 weeks | 
| Laboratory test (Urinalysis): Occult blood | Number and percentage of subjects will be presented. Shift tables before and after the dose will be presented. | Up to 8 weeks | 
| Vital signs: Systolic blood pressure (mmHg) | - Summary statistics, scattered plots before and after the dose will be presented. | Up to 8 weeks | 
| Vital signs: Diastolic blood pressure (mmHg) | - Summary statistics, scattered plots before and after the dose will be presented. | Up to 8 weeks | 
| Vital signs: Pulse rate (bpm) | - Summary statistics, scattered plots before and after the dose will be presented. | Up to 8 weeks | 
| Vital signs: Body temperature (°C) | - Summary statistics, scattered plots before and after the dose will be presented. | Up to 8 weeks | 
| Measured values and fluctuations in body weight | - Body weight | Up to 8 weeks | 
| ECG parameter: RR interval (msec) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| ECG parameter: PR interval (msec) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| ECG parameter: QRS interval (msec) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| ECG parameter: QT interval (msec) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| ECG parameter: QTcF interval (msec) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| ECG parameter: Pulse rate (bpm) | - Summary statistics will be presented for measured values and changes from baseline. | Up to 8 weeks | 
| KSP-0243 concentration in plasma at each time point | - KSP-0243 concentration in plasma | Up to 8 weeks | 
| KSP-0243 concentration in colorectal mucosa at Week 8 | - KSP-0243 concentration in colorectal mucosa | Up to 8 weeks |