COMPARE - Pediatric Inflammatory Bowel Disease (PIBD)

Measure of Crohn's disease (CD) impact on patients' daily lives (TUMMY CD)

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

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.

Measures of ulcerative colitis (UC) impact on patients' daily lives (TUMMY UC)

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

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.

PROMIS Symptom Measure of Irritable Bowel Disease (IBD)

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

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

Change in Pediatric PROMIS® Patient-Reported Outcome (PRO) Pain Interference Score for Crohn's Disease and Ulcerative Colitis (CD and UC)

Time frame: Baseline, months 6, 12, 18, 24, 30, 36

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.

Change in Pediatric PROMIS® Fatigue Score for Crohn's Disease and Ulcerative Colitis (CD and UC)

Time frame: Baseline, months 6, 12, 18, 24, 30, 36

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.

Change in Pediatric PROMIS® Anxiety Score for Crohn's Disease and Ulcerative Colitis (CD and UC)

Time frame: Baseline, months 6, 12, 18, 24, 30, 36

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.

Change in Pediatric PROMIS® Depression Score for Crohn's Disease and Ulcerative Colitis (CD and UC)

Time frame: Baseline, months 6, 12, 18, 24, 30, 36

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.

Change in sPCDAI Score for Crohn's Disease

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

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

Change in Pediatric Ulcerative Colitis Activity Index (PUCAI) Score

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

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.

What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.