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

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

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).

Study Overview

• Status: Completed
• Conditions: Crohn's Disease
• Intervention / Treatment: Drug: Ustekinumab

• Study Type: Observational
• Enrollment (Actual): 545

Contacts and Locations

Study Locations

• United States
  • Vermont
    • Burlington, Vermont, United States, 05408
      • ImproveCareNow, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population includes pediatric participants (age greater than or equal to [>=] 2 to less than [<] 18 years with body weight >=40 kilograms [kg]) and young adults (age >= 18 to < 26 years) with moderately to severely active Crohn's disease. The primary study population will be pediatric participants who have moderate to severe disease activity defined as short pediatric Crohn's disease activity index (sPCDAI) >= 30, are treated with ustekinumab, and are followed at an ImproveCareNow (ICN) center.

Description

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)

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Participants with Crohn's Disease (CD); 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; No interventions will be administered as a part of this study. Participants will receive ustekinumab as per their routine clinical practice.

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Janssen Research & Development, LLC
• Collaborators: Children's Hospital Medical Center, Cincinnati; ImproveCareNow (ICN)
• Investigators: Study Director: Janssen Research & Development, LLC Clinical Trial, Janssen Research & Development, LLC

Study record dates

Study Major Dates

• Study Start (Actual): March 21, 2022
• Primary Completion (Actual): May 22, 2023
• Study Completion (Actual): May 22, 2023

Study Registration Dates

• First Submitted: February 15, 2022
• First Submitted That Met QC Criteria: February 15, 2022
• First Posted (Actual): February 16, 2022

Study Record Updates

• Last Update Posted (Actual): May 13, 2025
• Last Update Submitted That Met QC Criteria: May 12, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Intestinal Diseases; Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease; Dermatologic Agents; Ustekinumab
• Other Study ID Numbers: CR109167; CNTO1275CRD3010 (Other Identifier: Janssen Research & Development, LLC)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at www.janssen.com/clinical-trials/transparency. As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at yoda.yale.edu

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes