PIBD-SETQuality; the PIBD-NET Inception Cohort and Safety Registry

Paediatric Inflammatory Bowel Diseases Network for Safety, Efficacy, Treatment and Quality Improvement of Care: The PIBD-NET Inception Cohort and Safety Registry

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.

Study Overview

• Status: Unknown
• Conditions: Inflammatory Bowel Diseases; Crohn Disease; Ulcerative Colitis
• Intervention / Treatment: Other: No intervention

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.

• Study Type: Observational
• Enrollment (Anticipated): 1000

Contacts and Locations

• Study Contact: Name: Frank Ruemmele, MD, PhD; Email: frank.ruemmele@nck.aphp.fr
• Study Contact Backup: Name: Lissy de Ridder, MD, PhD; Email: l.deridder@erasmusmc.nl

Study Locations

• Netherlands
  • Rotterdam, Netherlands, 3015GD
    • Recruiting
    • Erasmus MC - Sophia Children's Hospital
    • Contact: Lissy de Ridder, MD, PhD; Email: l.deridder@erasmusmc.nl
• United Kingdom
  • London, United Kingdom
    • Recruiting
    • Royal Hospital London
    • Contact: Nicholas Croft, MD, PhD; Email: n.m.croft@qmul.ac.uk

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 19 years (ADULT, CHILD)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Patients with paediatric-onset IBD (either Crohn's disease, ulcerative colitis or IBD-unclassified).

Description

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.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Predictors for outcome and treatment response or non-response	Factors predictive for outcome and treatment response or non-response This includes factors at baseline and 12 weeks after disease-onset that predict adverse outcomes (e.g. fibrostricturing disease, penetrating disease, active perianal fistula or abcess and need of surgery).	Over a period of three years after inclusion of the last patient.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
New biomarkers as classifiers of response or non-response to therapy	Part of the children in the inception cohort will deliver biological specimens to develop new biomarkers as classifiers of disease risk of complicated disease or side effects in PIBD patients, as well as predictors of (non-)response to a specific immunomodulator or biological agent.	Over a period of three years after inclusion of the last patient.
Identification of patients with rare and serious complications	Secondary outcome is to identify patients with rare and serious complications of PIBD or its treatment.	Until the end of patients recruitment, for a period of 2 years.

Collaborators and Investigators

• Sponsor: PIBD-Net
• Collaborators: European Commission
• Investigators: Principal Investigator: Lissy de Ridder, MD, PhD, Erasmus MC - Sophia Children's Hospital; Principal Investigator: Nicholas Croft, MD, PhD, Barts and the London School of Medicine, Queen Mary University of London

Publications and helpful links

General Publications

• 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.
• 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.
• 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.

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 3, 2017
• Primary Completion (ANTICIPATED): December 31, 2020
• Study Completion (ANTICIPATED): December 31, 2020

Study Registration Dates

• First Submitted: June 14, 2018
• First Submitted That Met QC Criteria: June 26, 2018
• First Posted (ACTUAL): June 27, 2018

Study Record Updates

• Last Update Posted (ACTUAL): March 6, 2020
• Last Update Submitted That Met QC Criteria: March 5, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Gastrointestinal Diseases; Gastroenteritis; Inflammatory Bowel Diseases; Crohn Disease; Intestinal Diseases
• Other Study ID Numbers: PIBD-Net 2016-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No