Colitis, Ulcerative
Conditions
Keywords
ulcerative, colitis, ulcerative colitis, etrasimod, pediatrics
Brief summary
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).
Interventions
DRUGEtrasimod
Once daily by mouth
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
Have a diagnosis of ulcerative colitis (UC) that is moderately to severely active Participants are permitted to be receiving a therapeutic dose of select UC therapies
Exclusion criteria
Severe extensive colitis Diagnosis of Crohn's disease (CD) or indeterminate colitis or the presence or history of a fistula consistent with CD Diagnosis of microscopic colitis, ischemic colitis, or infectious colitis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number and percent of enrolled participants with clinical remission based on Modified Mayo Score (MMS) at Week 52 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number and percent of enrolled participants PUCAI clinical response from baseline to Week 260 | Baseline, through 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. |
| Number and percentage of participants reporting a positive taste/palatability score | Week 2 | The responses to taste acceptability questionnaire on etrasimod tablets and granules will be summarized using count and percentage for SAS |
| Change from baseline in Z-Scores height and weight | Baseline through Week 260 | 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. |
| Number and percent of enrolled participants with clinical remission based on MMS at Week 12 | 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. |
| Number and percent of enrolled participants with clinical response based on MMS score components at Week 12 | 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. |
| Number and percent of enrolled participants with clinical response based on MMS score components at Week 52 | 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. |
| Number and percent of enrolled participants endoscopic improvement based on MMS score components at Week 12 | Week 12 | 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. |
| Number and percent of enrolled participants endoscopic improvement based on MMS score components at Week 52 | 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. |
| 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 | 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. |
| 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 | 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. |
| Number and percent of enrolled participants Symptomatic remission at all time points up to Week 52 | 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. |
| Number of Participants with Treatment Emergent Treatment-Related Adverse Events (AEs), including Serious Adverse Events (SAEs) and AEs leading to discontinuation. | Baseline up to 28 days after last dose of study intervention | 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. |
| Number of Participants with Clinically Significant Findings in Laboratory Examinations | Baseline up to 28 days after last dose of study intervention | 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. |
| Number of Participants with Clinically Significant Change in Vital Signs | Baseline up to week 260 | 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. |
| Number and percent of enrolled participants Pediatric Ulcerative Colitis Activity Index (PUCAI) clinical remission from baseline to Week 260 | Baseline, through 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. |
| Plasma concentration verses time of study intervention | Baseline, Weeks 2 and 4 | 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. |
Countries
Australia, Canada, France, Germany, Israel, Italy, Japan, Poland, United Kingdom, United States
Contacts
CONTACTPfizer CT.gov Call Center
STUDY_DIRECTORPfizer CT.gov Call Center
Pfizer
Outcome results
None listed