Clinical trial NCT03571373: PIBD-SETQuality: the Inception Cohort and Safety Registry
Official title: Paediatric Inflammatory Bowel Diseases Network for Safety, Efficacy, Treatment and Quality Improvement of Care: The PIBD-NET Inception Cohort and Safety Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Inflammatory Bowel Diseases
Crohn Disease
Ulcerative Colitis
Lead sponsor: PIBD-Net
Sponsor class: OTHER
Collaborators: name: European Commission
First posted: 2018-06-27
Enrollment: 1500
Sex: ALL
Minimum age (years): 0
Maximum age (years): 19
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with paediatric-onset IBD (either Crohn's disease, ulcerative colitis or IBD-unclassified).

Brief summary: The purpose of this study is to analyse effectiveness and safety signals of current treatment strategies in routine practice for patients with pediatric-onset inflammatory bowel disease (PIBD) and to correlate this to their individual risk factors.

Detailed description: In this prospective observational study children with newly diagnosed inflammatory bowel disease (IBD) will be included in the inception cohort. The primary aim of the inception cohort is to analyse the effectiveness and safety signals of current treatment strategies and to correlate them to individual risk factors. In order to capture information on rare and severe complications in PIBD as well, the safety registry was designed to estimate incidence and prevalence rates of these complications and get more insight in disease and treatment characteristics of these patients.

Eligibility criteria: Inclusion Criteria Inception cohort:

Newly diagnosed patient, \<18 years of age, with a likely diagnosis of IBD or a confirmed diagnosis of IBD can be included in the study. In order to be eligible to continue in the study the subject must meet all of the following criteria:

* Diagnosis is based on history, physical examination, laboratory, endoscopic, radiological and histological features according to the revised Porto criteria (1)
* Diagnosis has been made or is confirmed within 2 months of inclusion
* Data on all diagnostic procedures are available for inclusion in the database
* Informed consent of patient (if indicated) and parents has been obtained
* Concerning the patients of whom biological specimens will be included: patients have not started IBD treatment yet

Inclusion Criteria Safety Registry:

Any child with IBD \<19 years old with complications as detailed in the agreed safety monitoring list (or future updates of the list of conditions) can be reported. For the initial reporting of incident cases no patient identifiable details will be required.

Exclusion Criteria Inception cohort:

* Inability to read and understand the patient and family information sheets (for example insufficient knowledge of national language, where no health advocate or family member is available to translate and ensure full understanding of the study)
* Informed consent of patient or parents has not been obtained when required
* Patients on similar treatments as for IBD but for other conditions, or known with conditions directly affecting the IBD (e.g. immunodeficiency or major gastrointestinal resections)

Exclusion Criteria Safety registry: none.

Primary outcomes:
measure: Predictors for outcome and treatment response or non-response
timeFrame: Over a period of three years after inclusion of the last patient.
Secondary outcomes:
measure: New biomarkers as classifiers of response or non-response to therapy
timeFrame: Over a period of three years after inclusion of the last patient.
measure: Identification of patients with rare and serious complications
timeFrame: Until the end of patients recruitment, for a period of 2 years.
Other outcomes:
Locations:
city: Rotterdam
country: Netherlands
facility: Erasmus MC - Sophia Children's Hospital
city: London
country: United Kingdom
facility: Royal Hospital London
Study officials:
affiliation: Erasmus MC-Sophia Children's Hospital
name: Lissy de Ridder, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Barts and the London School of Medicine, Queen Mary University of London
name: Nicholas Croft, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Timmer A, Behrens R, Buderus S, Findeisen A, Hauer A, Keller KM, Kliemann G, Lang T, Lohr W, Rzehak P, Koletzko S; CEDATA-GPGE Study Group. Childhood onset inflammatory bowel disease: predictors of delayed diagnosis from the CEDATA German-language pediatric inflammatory bowel disease registry. J Pediatr. 2011 Mar;158(3):467-473.e2. doi: 10.1016/j.jpeds.2010.09.014. Epub 2010 Nov 4.
citation: De Greef E, Hoffman I, Smets F, Van Biervliet S, Bontems P, Hauser B, Paquot I, Alliet P, Arts W, Dewit O, De Vos M, Baert F, Bossuyt P, Rahier JF, Franchimont D, Vermeire S, Fontaine F, Louis E, Coche JC, Veereman G; IBD working group of BESPGHAN, BIRD. Paediatric Crohn Disease: Disease Activity and Growth in the BELCRO Cohort After 3 Years Follow-up. J Pediatr Gastroenterol Nutr. 2016 Aug;63(2):253-8. doi: 10.1097/MPG.0000000000001132.
citation: Aardoom MA, Kemos P, Tindemans I, Aloi M, Koletzko S, Levine A, Turner D, Veereman G, Neyt M, Russell RK, Walters TD, Ruemmele FM, Samsom JN, Croft NM, de Ridder L; PIBD-SETQuality consortium and PIBD-NET. International prospective observational study investigating the disease course and heterogeneity of paediatric-onset inflammatory bowel disease: the protocol of the PIBD-SETQuality inception cohort study. BMJ Open. 2020 Jul 1;10(7):e035538. doi: 10.1136/bmjopen-2019-035538.