COMPARE - Pediatric Inflammatory Bowel Disease (PIBD)

NCT07198113 | Recruiting

• 2 other identifiers: 25-2019Pro00118431
• Study Type: observational
• Target: 1,100
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Ulcerative Colitis, Pediatric; Inflammatory Bowel Diseases; Crohn Disease

Keywords

Ulcerative colitis, pediatric; Inflammatory bowel disease, pediatric; IBD; CD; UC; PIBD; Pediatrics; Children; Anti-TNF; Vedolizumab; Ustekinumab; Risankizumab; Guselkumab; Mirikizumab; Tofacitinib; Upadacitinib; Patient-reported outcomes (PROs); Tumor necrosis factor antagonists; IL-23 agent; PCORI; PCORnet; DCRI; Duke; UNC; University of North Carolina; Observational cohort; Anti-tumor necrosis factor (TNF) refractory; Comparative Effectiveness Research (CER); COMPARE

Outcome Measures

Primary Outcomes (9)

• 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.

  Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 36

• 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.

  Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 36

• 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

  Baseline, months 2, 4, 6, 8, 10, 12, 18, 24, 30, 36

• 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.

  Baseline, months 6, 12, 18, 24, 30, 36

• Change in Pediatric PROMIS® Fatigue 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 fatigue 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.

  Baseline, months 6, 12, 18, 24, 30, 36

• Change in Pediatric PROMIS® Anxiety 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 anxiety 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.

  Baseline, months 6, 12, 18, 24, 30, 36

• Change in Pediatric PROMIS® Depression 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 depression 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.

  Baseline, months 6, 12, 18, 24, 30, 36

• Change in sPCDAI Score for Crohn's Disease

  The short Pediatric Crohn's Disease Activity Index (sPCDAI) is a validated tool to assess disease activity in children with Crohn's disease. It is based on clinical symptoms, physical examination findings, and laboratory values. Scores range from 0 to 90, with higher scores indicating greater disease activity. The outcome will be measured as the mean change in sPCDAI score from baseline to follow-up. T Scores ≤15 indicate remission, \> 15-29 mild disease and ≥30 indicate moderate to severe disease

  Each clinic visit (baseline through follow-up, up to 3 years)

• Change in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score

  The Pediatric Ulcerative Colitis Index (PUCAI) is a patient-centered, 6-item measure assessing abdominal pain, stool characteristics, and patient functioning. Scores ranging from 0 - 85 with higher scores indicate more disease activity. A cut-point of \<10 indicates remission, ≥10 to 34 mild disease, ≥35-65 moderate disease, and ≥65 severe disease. Response is defined as a reduction of ≥ 20 points.

  Each clinic visit (baseline through follow-up, up to 3 years)

Secondary Outcomes (4)

• Change in Fecal Calprotectin Concentration (CD and UC)

  12 months after after taking prescribed medication for CD or UC

• Number of Participants Experiencing Adverse Events (CD and UC)

  After medication administration and every 3 months, up to 3 years

• Proportion of Participants Experiencing Adverse Events (CD and UC)

  After medication administration and every 3 months, up to 3 years

• Exploration of Heterogeneity of Treatment Effects (HTE) Across Clinical Subgroups (CD and UC)

  Baseline through follow -up for up to 3 years

Study Arms (3)

Children with Crohn's disease (CD)

Pediatric CD patients initiating non-anti-TNF biologics and small molecules that are FDA-approved for adult populations

Children with Ulcerative Colitis (UC)

Pediatric UC patients initiating non-anti-TNF biologics and small molecules that are FDA-approved for adult populations

Retrospective Safety Analysis

Retrospective cohort focused on the long-term safety of non-anti-TNF biologics and small molecules that are FDA-approved for adult populations in for CD and or UC. Electronic health record (EHR) data (retrospective cohort) will be collected from medical charts and EHRs at participating institutions.

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children with Crohn's disease (CD) or ulcerative colitis (UC) whose disease is refractory to anti-TNF therapy

You may qualify if:

• 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.
• Ability to provide child assent, if required per regulatory or local institutional guidelines, and parental informed consent in English or Spanish

You may not qualify if:

• 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
• Patients with current ostomy

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• Patient-Centered Outcomes Research Institute: collaborator
• Duke Clinical Research Institute: collaborator

Study Sites (4)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Atrium Health

Charlotte, North Carolina, 28203, United States

RECRUITING

Duke Health System

Durham, North Carolina, 27701, United States

RECRUITING

University of Pittsburgh Medical Center, Children's Hospital

Pittsburgh, Pennsylvania, 15222, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Optional biospecimens will be collected throughout this study for future, unspecified research and may include blood, saliva, stool, and tissue samples.

MeSH Terms

Conditions

Pediatric ulcerative colitis; Inflammatory Bowel Diseases; Crohn Disease

Condition Hierarchy (Ancestors)

Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Michael D Kappelman, MD

  Universty of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Duke Clinical Research Institute COMPARE Call Center

CONTACT

833-850-2848; COMPARE@dm.duke.edu

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2025
• First Posted: September 30, 2025
• Study Start: November 1, 2025
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: April 17, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: beginning 9 and continuing for 36 months following publication
• Access Criteria: Investigator has approved IRB, IEC, or REB and an executed data use/sharing agreement with UNC.

Locations

US (4)