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

PIs:

Steve Steiner, MD, REALITI Co-Principal Investigator, Riley Children’s Hospital 

Shehzad Saeed, MD, REALITI Co-Principal Investigator, ImproveCareNow & Dayton Children’s Hospital 

Jeremy Adler, MD, REALITI Co- Principal Investigator, University of Michigan – C.S. Mott Children’s Hospital

Purpose: The primary objective of this study is to evaluate the clinical effectiveness of ustekinumab by determining the clinical remission rate in pediatric patients with moderately to  severely active Crohn’s Disease (CD) in the ICN registry who were treated with ustekinumab.

Primary Outcome: This study will generate knowledge about the use of ustekinumab in the management of pediatric Crohn’s Disease. The results of this study will be included as part of a Janssen submission to the FDA for the approval of the use of ustekinumab in pediatric patients, as they plan a phase 3 clinical trial of ustekinumab. In addition, the inclusion of RWE from ICN could help facilitate the approval of ustekinumab and other  new  drugs  for  IBD,  making  new  IBD treatments accessible  to  pediatric  patients sooner, enabling better outcomes for our patients. If the ICN-Janssen REALITI Study is successful, other new IBD drugs could be studied in a similar way, having a major impact on the health of our patients as well as on the sustainability of ICN.

Funding Source: Janssen has provided funding to ICN to perform this study; however, it is important to note that this  is  an  observational study with  a  retrospective  chart  review  and not an interventional study.

Study Period: The study will take approximately 9 to 18 months to complete from implementation to publication.

Recruitment Status: All eligible patients have been identified via the ICN  Registry, the 72 ICN centers with eligible patients have been invited to participate.

Contact: [email protected]

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