Therapeutic Drug Monitoring for Biological Therapy in Pediatric Inflammatory Bowel Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Ulcerative Colitis (UC), IBD-unclassified (IBD-U). Basic parameters: 0 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Austria
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Therapeutic Drug Monitoring for Biological Therapy in Pediatric Inflammatory Bowel Disease: a Prospective Non-interventional Multicentric Study
Overview
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.
Primary outcome measures
- Pharmacokinetic Evaluation of Plasma Level of Biological Therapy [Time frame: 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 outcome measures (3)
- Time Course of Disease Activity (clinical assessments) [Time frame: 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]
- Time course of Disease Activity (laboratory assessments) [Time frame: 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]
- Development of anti-drug antibodies during Biological Therapy [Time frame: 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.]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Austria · 10 centers
- Landeskrankenhaus Feldkirch — Feldkirch
- Universitätsklinikum Graz / Medizinische Universität Graz — Graz
- Medical University of Innsbruck — Innsbruck
- Klinikum Klagenfurt — Klagenfurt
- Kepler Universitätsklinikum Linz — Linz
- Paracelsus Medizinische Privatuniversität — Salzburg
- St. Anna Kinderspital — Vienna
- Universitätsklinik für Kinder- und Jugendheilkunde — Vienna
- … and 2 more centers
Identifiers
NCT: NCT06954311 · TDMpIBD (1126/2024)