The purpose of this study is to determine the safety, efficacy, and pharmacokinetics (PK) of etrasimod for the treatment of moderately to severely active ulcerative colitis in pediatrics participants (≥ 2 years up to \< 12 years of age). Participants who will complete the total 52-week treatment period will have the opportunity to continue in a Long-Term Extension (LTE) Period of up to 4 years (5 years after study enrollment).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Once daily by mouth
CHU de Québec - Université Laval
Québec, Quebec, Canada
RECRUITINGUniversitätsklinikum Leipzig
Leipzig, Saxony, Germany
NOT_YET_RECRUITINGUniversitaetsklinikum Tuebingen
Number and percent of enrolled participants with clinical remission based on Modified Mayo Score (MMS) at Week 52
Clinical remission was defined as a total Mayo score of 2 points or lower, with no individual subscore exceeding 1 point (SF= 1 or 0), ES=1 or 0 and RB=0. Mayo score: instrument designed to measure disease activity of ulcerative colitis. will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 52
Number and percent of enrolled participants with clinical remission based on MMS at Week 12
Clinical remission was defined as a total Mayo score of 2 points or lower, with no individual subscore exceeding 1 point (SF= 1 or 0), ES=1 or 0 and RB=0. Mayo score: instrument designed to measure disease activity of ulcerative colitis. will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 12
Number and percent of enrolled participants with clinical response based on MMS score components at Week 12
Clinical response was defined as a ≥2-point and ≥30% decrease from baseline in MMS, and a ≥1-point decrease from baseline in RB subscore or an absolute RB subscore ≤ 1. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 12
Number and percent of enrolled participants with clinical response based on MMS score components at Week 52
Clinical response was defined as a ≥2-point and ≥30% decrease from baseline in MMS, and a ≥1-point decrease from baseline in RB subscore or an absolute RB subscore ≤ 1. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 52
Number and percent of enrolled participants endoscopic improvement based on MMS score components at Week 12
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Tübingen, Germany
Saitama Prefectural Children's Medical Center
Saitama-shi, Saitama, Japan
RECRUITINGJapanese Red Cross Kumamoto Hospital
Kumamoto, Japan
RECRUITINGJuntendo University Hospital
Tokyo, Japan
RECRUITINGCentrum Zdrowia MDM
Warsaw, Masovian Voivodeship, Poland
NOT_YET_RECRUITINGMedical Network Spółka z o.o. WIP Warsaw IBD Point Profesor Kierkuś
Warsaw, Masovian Voivodeship, Poland
RECRUITINGGyncentrum sp. z o.o. NZOZ Holsamed - oddział Libero
Katowice, Poland
NOT_YET_RECRUITINGEndoscopic improvement defined as ES ≤1 (excluding friability). These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 12
Number and percent of enrolled participants endoscopic improvement based on MMS score components at Week 52
Endoscopic improvement defined as ES ≤1 (excluding friability). These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 52
Number and percent of enrolled participants Clinical remission at Week 12 and who had not been receiving corticosteroids for ≥2 weeks immediately prior to Week 12
Clinical remission was defined as a total Mayo score of 2 points or lower, with no individual subscore exceeding 1 point (SF= 1 or 0), ES=1 or 0 and RB=0. Number of weeks off corticosteroids prior to week 12 visit will be used to determine end point. Mayo score: instrument designed to measure disease activity of ulcerative colitis. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 12
Number and percent of enrolled participants Clinical remission at Week 52 and who had not been receiving corticosteroids for ≥12 weeks immediately prior to Week 52
Clinical remission was defined as a total Mayo score of 2 points or lower, with no individual subscore exceeding 1 point (SF= 1 or 0), ES=1 or 0 and RB=0. Number of weeks off corticosteroids prior to week 52 visit will be used to determine end point. Mayo score: instrument designed to measure disease activity of ulcerative colitis. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 52
Number and percent of enrolled participants Symptomatic remission at all time points up to Week 52
Symptomatic remission was defined as SF subscore = 0 or 1 and an RB subscore = 0. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Week 52
Number and percent of enrolled participants Pediatric Ulcerative Colitis Activity Index (PUCAI) clinical remission from baseline to Week 260
Clinical remission by PUCAI is defined as a score \<10. These assessments will be conducted at each visit. These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Baseline, through Week 260
Number and percent of enrolled participants PUCAI clinical response from baseline to Week 260
Clinical response by PUCAI is defined as a score ≥ 20 point reduction from baseline. These assessments will be conducted at each visit.These results will be summarized by the number and percentage of participants achieving the response, along with a two-sided 95% CI.
Time frame: Baseline, through Week 260
Number and percentage of participants reporting a positive taste/palatability score
The responses to taste acceptability questionnaire on etrasimod tablets and granules will be summarized using count and percentage for SAS
Time frame: Week 2
Number and percentage of participants reporting a positive taste/palatability score
The responses to taste acceptability questionnaire on etrasimod tablets and granules will be summarized using count and percentage for SAS
Time frame: Week 12
Change from baseline in Z-Scores height and weight
These assessments will be conducted at each visit. The values and change from baseline will be summarized using number of observations, mean, standard deviation, minimum and maximum values by visit for SAS.
Time frame: Baseline through Week 260
Number of Participants with Treatment Emergent Treatment-Related Adverse Events (AEs), including Serious Adverse Events (SAEs) and AEs leading to discontinuation.
Treatment-related AE was any untoward medical occurrence attributed to study drug in a participant who received study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. Relatedness to etrasimod was assessed by the investigator (Yes/No). Participants with multiple occurrences of an AE within a category were counted once within the category.
Time frame: Baseline up to 28 days after last dose of study intervention
Number of Participants with Clinically Significant Findings in Laboratory Examinations
Following parameters were analyzed for laboratory examination: hematology (hemoglobin, hematocrit, red blood cell count, platelet count, white blood cell count, total neutrophils, eosinophils, monocytes, basophils, lymphocytes); Hepatobiliary biochemistry: Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Albumin, Alkaline Phosphatase, Total Bilirubin ; Renal Function Tests: Blood Urea Nitrogen (BUN), Creatinine, Creatinine Kinase, Uric Acid ; Electrolytes: Sodium, Potassium; Glucose; Urine analysis: (decimal logarithm of reciprocal of hydrogen ion activity )\[pH\], Specific gravity. Clinically significant laboratory abnormality findings were based on investigator discretion.
Time frame: Baseline up to 28 days after last dose of study intervention
Number of Participants with Clinically Significant Change in Vital Signs
Following vital sign parameters were assessed: diastolic blood pressure, systolic blood pressure, respiration rate, pulse rate, temperature and body weight. Number of participants with clinically significant change in any vital sign parameter compared to baseline were reported. Clinically significant change in vital signs criteria were based on investigator's discretion.
Time frame: Baseline up to week 260
Plasma concentration verses time of study intervention
Samples collected prior to daily dosing of study intervention for measurement of plasma concentrations of etrasimod will be analyzed using a validated analytical method in compliance with applicable SOPs.
Time frame: Baseline, Weeks 2 and 4