A Study of Ustekinumab Treatment in Children With Crohn's Disease

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the effectiveness of ustekinumab in achieving clinical remission in pediatric participants (greater than or equal to [>=] 2 to less than [<] 18 years and weight >= 40 kilograms [kg] at baseline).

Official Title
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Real-world Evidence for the Effectiveness and Safety of Ustekinumab Treatment in Children With Crohn's Disease: A Retrospective Cohort Study Using the ImproveCareNow Registry Data

Conditions
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Crohn's Disease

Intervention / Treatment
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* Drug: Ustekinumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Having at least one ICN visit with documented new use of ustekinumab Participants with a documented Crohn's disease (CD) diagnosis at the time ustekinumab was initiated (that is, Baseline); If diagnosis at baseline is missing, then the diagnosis from previous visit within study window will be utilized Having at least one ICN visit prior to the ICN visit when ustekinumab is first documented Having received first dose of ustekinumab on or before June 22, 2019 Having provided informed consent for use of ICN data for research purposes Exclusion Criteria: Documented exposure to ustekinumab before enrollment in ICN (by chart review)

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Participants with Crohn's Disease (CD)<br>Participants with moderate-to-severe active CD enrolled in ImproveCareNow (ICN) pediatric Inflammatory bowel disease (IBD) registry will be observed who are being treated with ustekinumab under real world setting and will be divided into 9 cohorts. Cohorts 1-6 for age group >=2 to <18 years (pediatric), cohorts 7-9 for young adults within age group >=18 to <26 years. Cohort 1: participants with moderate-severe CD and body weight >=40 kg, cohort 2: participants with moderate-severe CD and body weight <40 kg, cohort 3: all participants with moderate-severe CD, cohort 4: participants with active disease and body weight >=40 kg, cohort 5: participants with active disease and body weight <40 kg, cohort 6: all participants treated with ustekinumab, cohort 7: young adults with moderate-severe CD, cohort 8: young adults with active-disease; and cohort 9: all young adults treated with ustekinumab. Only data available per routine clinical practice will be collected within this study. | Drug: Ustekinumab<br>* No interventions will be administered as a part of this study. Participants will receive ustekinumab as per their routine clinical practice.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cohort 1: Percentage of Pediatric Participants with Clinical Remission at Week 52 | Percentage of Pediatric participants with clinical remission at Week 52 will be reported. Clinical remission is defined by short pediatric Crohn's disease activity index (sPCDAI) less than or equal to (<=) 10. | Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cohort 7: Percentage of Young Adult Participants with Clinical Remission at Week 52 | Percentage of young adult participants with clinical remission at Week 52 will be reported. | Week 52 | 
| Cohorts 2, 3, 4, 5, 6, 8 and 9: Percentage of Participants with Clinical Remission at Week 52 | Percentage of participants with clinical remission at Week 52 will be reported. | Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 Evaluated by Ustekinumab Dose Bands | Percentage of participants with clinical remission at Week 52 evaluated by ustekinumab dose bands will be reported. | Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 with No Corticosteroid Use | Percentage of participants with clinical remission at Week 52 with no corticosteroid use will be reported. | Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 by PGA Score | Percentage of participants with clinical remission at Week 52 by Physician Global Assessment (PGA) score will be reported. | Week 52 | 
| Cohorts 1 and 7: Change from Baseline in Height at Week 52 | Change from baseline in height at Week 52 will be reported. | Baseline and Week 52 | 
| Cohorts 1 and 7: Change from Baseline in Weight at Week 52 | Change from baseline in weight at Week 52 will be reported. | Baseline and Week 52 | 
| Cohorts 1 and 7: Change from Baseline in Body Mass Index (BMI) at Week 52 | Change from baseline in BMI (measured with age and sex-specific z-scores) at Week 52 will be reported. | Baseline and Week 52 | 
| Cohorts 1 and 7: Percentage of Participants Who Achieve Endoscopic Remission with SEMA-CD Score of 0 or 1 at Week 52 | Percentage of participants who achieve endoscopic remission with simplified endoscopic mucosal assessment for Crohn's disease (SEMA-CD) score of 0 or 1 at Week 52 will be reported. Endoscopic remission is defined as SEMA-CD score of 0 or 1. SEMA-CD mucosal disease activity score is defined as inactive = 0; minimal = 1; mild = 2-4; moderate = 5-9, or severe = greater than or equal to (>=) 10. | Week 52 | 
| Cohorts 1 and 7: Change from Baseline to Week 52 in Mucosal Disease Activity Measured with SEMA-CD Score | Change from baseline to Week 52 in mucosal disease activity measured with SEMA-CD score will be reported. SEMA-CD mucosal disease activity score is defined as inactive = 0; minimal = 1; mild = 2-4; moderate = 5-9, or severe = greater than or equal to (>=) 10. | Baseline to Week 52 | 
| Cohorts 1 and 7: Percentage of Participants that Discontinued Ustekinumab Use Among Participants who Initiated Ustekinumab Therapy at Week 52 | Percentage of participants that discontinued ustekinumab use among participants who initiated ustekinumab therapy at Week 52 will be reported. | Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Occurrence of Inflammatory Bowel Diseases (IBD)-related Hospitalizations Who had Initiated Ustekinumab Therapy | Percentage of participants with occurrence of IBD-related hospitalizations who had initiated ustekinumab therapy will be reported. | Up to Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Occurrence of IBD-related Surgeries Who had Initiated Ustekinumab Therapy | Percentage of participants with occurrence of IBD-related surgeries who had initiated ustekinumab therapy will be reported. | Up to Week 52 | 
| Cohorts 1 and 7: Percentage of Participants with Adverse Events of Special Interest (AESI) Who had Initiated Ustekinumab Therapy | Percentage of participants with AESIs (example: malignancies, serious and opportunistic infection, anaphylaxis requiring discontinuation of ustekinumab) who had initiated ustekinumab therapy will be reported. | Up to Week 52 |