Clinical trial NCT06954311: Therapeutic Drug Monitoring for Biological Therapy in Pediatric Inflammatory Bowel Disease
Official title: Therapeutic Drug Monitoring for Biological Therapy in Pediatric Inflammatory Bowel Disease: a Prospective Non-interventional Multicentric Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Ulcerative Colitis (UC)
IBD-unclassified (IBD-U)
Keywords: IBD
Pediatric
Biologicals
Infliximab
Adalimumab
Vedolizumab
Ustekinumab
Lead sponsor: Medical University Innsbruck
Sponsor class: OTHER
First posted: 2025-05-01
Enrollment: 120
Sex: ALL
Minimum age (years): 0
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric patients with IBD under 18 years of age (Crohn's disease, ulcerative colitis, and IBD-unclassified) with Treatment withInfliximab, Adalimumab, Vedolizumab, or Ustekinumab during induction or maintenance phase.

Brief summary: Inflammatory Bowel Diseases (IBD), which include Crohn's disease (CD), ulcerative colitis (UC), and the unclassified form referred to as indeterminate colitis, are most commonly diagnosed during adolescence and early adulthood. In recent decades, an increasing incidence of IBD has been observed in this age group.

A wide range of immunomodulatory agents, from corticosteroids to monoclonal antibodies, are now available for the treatment of IBD. These antibodies, known as biologics, target, for example, tumor necrosis factor-alpha (TNF-α; e.g., infliximab and adalimumab), integrin α4β7 (vedolizumab), or interleukin-12/23 (ustekinumab). While infliximab and adalimumab are approved for pediatric use in CD and UC, vedolizumab is only approved for moderate-to-severe UC from the age of 16, and ustekinumab is not approved for pediatric use at all. Nevertheless, vedolizumab and ustekinumab are frequently used off-label in cases of treatment failure with approved therapies, as efficacy has been demonstrated in adult IBD patients, and since 2015, increasing pediatric literature has emerged on their use.

To facilitate appropriate dose adjustment in pediatric clinical practice, biologic therapies can be monitored through measurement of drug trough levels. Current pediatric guidelines already recommend incorporating therapeutic drug monitoring (TDM) of infliximab and adalimumab in the management of CD and UC. Studies on TDM for vedolizumab and ustekinumab have so far been conducted almost exclusively in adult IBD patients, where improved treatment responses have also been demonstrated.

The presented research is a prospective, non-interventional observational study involving pediatric IBD patients at multiple Austrian pediatric gastroenterology centers. The study duration is five years. The aim is to include at least 40 patients receiving induction and maintenance therapy with infliximab or adalimumab, and 20 patients treated with vedolizumab or ustekinumab during both treatment phases. The primary objective is to gain a better understanding of the pharmacokinetic dynamics of these biologics and the associated treatment response in pediatric settings. Data will be collected exclusively from routine clinical assessments. No additional study-related visits or interventions are planned.


Eligibility criteria: Inclusion Criteria:

* Pediatric patients with an Inflammatory Bowel Disease (Crohn's disease, ulcerative colitis, and IBD-unclassified) being under 18 years of age
* Treatment with Infliximab, Adalimumab, Vedolizumab, or Ustekinumab during induction or maintenance phase

Exclusion Criteria:

-- Patients with primary (congenital) immunodeficiency

Primary outcomes:
measure: Pharmacokinetic Evaluation of Plasma Level of Biological Therapy
timeFrame: Plasma Levels will be assessed at Baseline and approximately at Weeks 2, 6, 14, 22, 30, 38, 46, and 54 during routine follow-up visits. Actual timing may vary slightly due to the non-interventional nature of the study.
Secondary outcomes:
measure: Time Course of Disease Activity (clinical assessments)
timeFrame: Clinical outcome scores will be assessed at Baseline and approximately at Weeks 2, 6, 14, 22, 30, 38, 46, and 54 during routine follow-up visits. Actual timing may vary slightly due to the non-interventional nature of the study
measure: Time course of Disease Activity (laboratory assessments)
timeFrame: Calprotectin will be assessed at Baseline and approximately at Weeks 2, 6, 14, 22, 30, 38, 46, and 54 during routine follow-up visits. Actual timing may vary slightly due to the non-interventional nature of the study
measure: Development of anti-drug antibodies during Biological Therapy
timeFrame: Anti-drug antibody Levels will be assessed at Baseline and approximately at Weeks 2, 6, 14, 22, 30, 38, 46, and 54 during routine follow-up visits. Actual timing may vary slightly due to the non-interventional nature of the study.
Other outcomes:
Locations:
city: Feldkirch
country: Austria
facility: Landeskrankenhaus Feldkirch
city: Graz
country: Austria
facility: Universitätsklinikum Graz / Medizinische Universität Graz
city: Innsbruck
country: Austria
facility: Medical University of Innsbruck
city: Klagenfurt
country: Austria
facility: Klinikum Klagenfurt
city: Linz
country: Austria
facility: Kepler Universitätsklinikum Linz
city: Salzburg
country: Austria
facility: Paracelsus Medizinische Privatuniversität
city: Villach
country: Austria
facility: Landeskrankenhaus Villach
city: Wien
country: Austria
facility: St. Anna Kinderspital
city: Wien
country: Austria
facility: Universitätsklinik für Kinder- und Jugendheilkunde
city: Wien
country: Austria
facility: Klinik Donaustadt
Study officials:
References: