COMPARE - Pediatric Inflammatory Bowel Disease (PIBD)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age1-18
SponsorUniversity of North Carolina, Chapel Hill
About this trial
The purpose of the study is to compare the clinical effectiveness and safety of newer inflammatory bowel disease (IBD) medications in anti-tumor necrosis factor (TNF) refractory patients with pediatric IBD (PIBD). Refractory means that there was no clinical response to anti-tumor necrosis factor (TNF) drugs or that the if there was a response, it is no longer present. The main question this study aims to answer is:
Are the newer medications used to treat IBD just as safe and effective for treating IBD in children.
Participants will already be taking these newer medications as assigned by their regular health care provider.Participants' care will be managed by their regular healthcare provider as part of usual (standard) care for those with PIBD. While taking these medications, participants will be asked to answer questions about their symptoms and health periodically over the course of the study.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age < 18 years at study enrollment
Diagnosis of CD, UC, or IBD-U by standard diagnostic criteria
Prior non-response or loss of response to one or more anti-TNF agents
Planning to initiate treatment with any of the following comparator agents: vedolizumab (α4β7 integrin antibody), ustekinumab (anti-IL-12/23 antibody), risankizumab, guselkumab, or mirikizumab, (IL-23 inhibitors), tofacitinib (JAK inhibitor), and upadacitinib (JAK inhibitor). Biosimilars or generic medications for any of the above will also be allowed and handled/analyzed in an identical manner to originators.
Disqualifiers
Plans to change care to a different center within 1 year
Prior use of a comparator agent (i.e., only patients starting their first comparator medication as monotherapy following anti-TNF will be eligible)
Contraindication to any of the treatments under investigation
Patients with UC or IBD-U who have undergone colectomy
Trial population
Children with Crohn's disease (CD) or ulcerative colitis (UC) whose disease is refractory to anti-TNF therapy
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
1,100 Participants
are grouped into 3 trial groupsGroup A: Children with Crohn's disease (CD)
Group B: Children with Ulcerative Colitis (UC)
Group C: Retrospective Safety Analysis
Trial outcomes
Primary outcomes
1
Measure of Crohn's disease (CD) impact on patients' daily lives (TUMMY CD)
The TUMMY CD patient reported outcome (PRO) has been developed and is undergoing validation. For our CD cohort, we will collect data on both the TUMMY-CD and the PROMIS IBD PROs. If TUMMY-CD is proven to be a reliable and valid PRO by the time of our data analysis, we will utilize this as our primary PRO. Otherwise, we will rely on PROMIS IBD PROs. Change will be calculated as the difference between baseline and post-baseline scores, with higher scores reflecting greater symptom severity.
Time frame
Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 362
Measures of ulcerative colitis (UC) impact on patients' daily lives (TUMMY UC)
Description: The TUMMY UC patient reported outcome (PRO) is a validated patient-reported outcome tool designed to assess gastrointestinal symptom burden in children and adolescents with ulcerative colitis (UC). Change will be calculated as the difference between baseline and post-baseline scores, with higher scores reflecting greater symptom severity. Scores range from 0 to 114 with higher values indicating greater symptom burden.
Time frame
Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 363
PROMIS Symptom Measure of Irritable Bowel Disease (IBD)
NIH Patient Reported Outcome Measurement Information System (PROMIS) symptoms scale is a disease-specific PRO. Prior validation of the PROMIS IBD measure has demonstrated acceptable internal consistency (Cronbach's alpha of 0.74), as well as discriminative and known groups validity. The PROMIS IBD symptom score has been standardized such that a T score of 50 represents the median score in the reference pediatric IBD population. T scores range from 40 to 80 with higher scores indicating higher symptom burden and a minimal clinically important difference (MCID) of 5. We will not use the PROMIS IBD for measure of UC
Time frame
Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 364
Change in Pediatric PROMIS® Patient-Reported Outcome (PRO) Pain Interference Score for Crohn's Disease and Ulcerative Colitis (CD and UC)
Pediatric Patient Reported Outcome Measurement Information System (PROMIS) assesses domains relevant to children and adolescents with CD and UC and their reported pain score. PROMIS Pediatric PROs domains have been standardized such that a T score of 50 represents the median score in the reference pediatric IBD population with higher scores indicating higher symptom burden and minimal clinically important difference (MCID) of 5.
Time frame
Baseline, months 6, 12, 18, 24, 30, 36Secondary outcomes
1
Change in Fecal Calprotectin Concentration (CD and UC)
Fecal calprotectin is a noninvasive biomarker of intestinal inflammation. Stool samples will be collected at baseline and follow-up visits. Calprotectin concentration will be measured in micrograms per gram (µg/g) of stool. The outcome will be measured as the mean change in fecal calprotectin from baseline to follow-up. Higher values indicate greater intestinal inflammation. A threshold of \< 150 μg/g will be used to define remission.
Time frame
12 months after after taking prescribed medication for CD or UC2
Number of Participants Experiencing Adverse Events (CD and UC)
Safety will be assessed by collecting and monitoring all adverse events (AEs) and serious adverse events (SAEs). Adverse events will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), where applicable. The outcome will be summarized as the number of participants experiencing AE/SAE of grade 2 or higher.
Time frame
After medication administration and every 3 months, up to 3 years3
Proportion of Participants Experiencing Adverse Events (CD and UC)
Safety will be assessed by collecting and monitoring all adverse events (AEs) and serious adverse events (SAEs). Adverse events will be graded according to the Common Terminology Criteria for Adverse Events (CTCAE), where applicable. The outcome will be summarized as the proportion of participants experiencing AE/SAE of grade 2 or higher.
Time frame
After medication administration and every 3 months, up to 3 years4
Exploration of Heterogeneity of Treatment Effects (HTE) Across Clinical Subgroups (CD and UC)
Heterogeneity of treatment effects (HTE) will be evaluated per baseline phenotype query that will run against the electronic health record (EHR) at sites. Treatment effects will be estimated within each subgroup (CD and UC) and compared to assess differential response patterns. This analysis is intended to identify variability in treatment response.
Time frame
Baseline through follow -up for up to 3 yearsSponsors and contacts
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University of North Carolina, Chapel Hill
Lead sponsor
Patient-Centered Outcomes Research Institute
Collaborator
Duke Clinical Research Institute
Collaborator
Source ClinicalTrials.gov