Novel Agents on the Therapeutic Horizon For Paediatric Inflammatory Bowel Diseases: An Analysis of Clinical Trials Registries

Abstract

Despite considerable advancements in the therapeutic landscape of inflammatory bowel disease (IBD) for adults, long delays exist for paediatric IBD (pIBD) approval, with a median delay of >7 years following adult approval. Our aim is to summarise the landscape of pIBD clinical trials through a review of trial registries. We conducted a cross-sectional review of ClinicalTrials.gov and ClinicalTrialsRegister.eu to identify investigational and approved therapeutic agents for the treatment of pIBD. All interventional studies involving pIBD (aged <18 years) from database inception to July 2, 2026, were considered for inclusion. Of 3941 records screened, 123 completed trials (77 randomised controlled trials [RCTs] and 46 non-RCTs) and 90 active trials (55 RCTs and 35 non-RCTs) met the inclusion criteria. A majority of completed trials (71%) recruited mixed adult and paediatric populations, whereas nearly half of the active trials recruited exclusively paediatric patients, with most studies enrolling participants aged 2–17 years. Biologics were assessed in 81 trials (42 completed and 39 active), including the approved agents (n = 39), infliximab, adalimumab, golimumab, and ustekinumab, and investigational therapies (n = 42) such as vedolizumab, guselkumab, mirikizumab, risankizumab, duvakitug, afimkibart, and tulisokibart. Nine completed and 11 active trials assess small molecule therapies, including tofacitinib, upadacitinib, filgotinib, etrasimod, ozanimod, and obefazimod. Efforts to expedite approval of new agents in pIBD are warranted to ensure timely access to effective medications. Consideration for novel trial designs alongside continued engagement with regulatory bodies, sponsors, and the academic pIBD community is essential to advance drug approvals for pIBD.