Clinical trial NCT05242458: A Study of Ustekinumab Treatment in Children With Crohn's Disease
Official title: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Crohn's Disease
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
Collaborators: name: ImproveCareNow (ICN)
name: Children's Hospital Medical Center, Cincinnati
First posted: 2022-02-16
Enrollment: 545
Sex: ALL
Minimum age (years): 2
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
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.

Brief summary: 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).


Eligibility criteria: 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)

Primary outcomes:
measure: Cohort 1: Percentage of Pediatric Participants with Clinical Remission at Week 52
timeFrame: Week 52
Secondary outcomes:
measure: Cohort 7: Percentage of Young Adult Participants with Clinical Remission at Week 52
timeFrame: Week 52
measure: Cohorts 2, 3, 4, 5, 6, 8 and 9: Percentage of Participants with Clinical Remission at Week 52
timeFrame: Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 Evaluated by Ustekinumab Dose Bands
timeFrame: Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 with No Corticosteroid Use
timeFrame: Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Clinical Remission at Week 52 by PGA Score
timeFrame: Week 52
measure: Cohorts 1 and 7: Change from Baseline in Height at Week 52
timeFrame: Baseline and Week 52
measure: Cohorts 1 and 7: Change from Baseline in Weight at Week 52
timeFrame: Baseline and Week 52
measure: Cohorts 1 and 7: Change from Baseline in Body Mass Index (BMI) at Week 52
timeFrame: Baseline and Week 52
measure: Cohorts 1 and 7: Percentage of Participants Who Achieve Endoscopic Remission with SEMA-CD Score of 0 or 1 at Week 52
timeFrame: Week 52
measure: Cohorts 1 and 7: Change from Baseline to Week 52 in Mucosal Disease Activity Measured with SEMA-CD Score
timeFrame: Baseline to Week 52
measure: Cohorts 1 and 7: Percentage of Participants that Discontinued Ustekinumab Use Among Participants who Initiated Ustekinumab Therapy at Week 52
timeFrame: Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Occurrence of Inflammatory Bowel Diseases (IBD)-related Hospitalizations Who had Initiated Ustekinumab Therapy
timeFrame: Up to Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Occurrence of IBD-related Surgeries Who had Initiated Ustekinumab Therapy
timeFrame: Up to Week 52
measure: Cohorts 1 and 7: Percentage of Participants with Adverse Events of Special Interest (AESI) Who had Initiated Ustekinumab Therapy
timeFrame: Up to Week 52
Other outcomes:
Locations:
city: Burlington
country: United States
facility: ImproveCareNow, Inc.
state: Vermont
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References: