Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease

Chronic Granulomatous Disease Clinical Trial

Official title:

Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06162936
• Other study ID #: IBD20232024
• Status: Recruiting
• Start date: November 1, 2023
• Est. completion date: February 2025

Study information

• Verified date: November 2023
• Source: National Institute for Mother and Child Health Alessandrescu Rusescu
• Contact: Alexis Virgil Cochino, MD, PhD
• Phone: +40723193648
• Email: alexis_virgil[@]yahoo.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

ABSTRACT Introduction Residual or absent oxidase function in peripheral neutrophils may point to an inborn defect of neutrophil function - chronic granulomatous disease (CGD) - whereas low to normal oxidative burst capacity has been linked to variants in various members of the NADPH-complex.

Aims To assess the clinical value of routinely measuring oxidative burst activity of granulocytes in pediatric patients diagnosed with very early onset IBD (VEO-IBD) and late onset IBD.

Objectives To investigate possible correlations between neutrophil function and IBD disease activity and to inquire the presence of genetic variants in those with low to absent oxidative burst. To identify the rate of monogenic VEO-IBD in our cohort.

Materials and Methods The proposal constitutes a collaborative effort among Romanian pediatric tertiary care centers to examine the value of assessing neutrophil function in all pediatric IBD patients. Children aged <18 years diagnosed with Crohn's disease, ulcerative colitis or IBD-undetermined and age-matched healthy controls are recruited. A DHR flow cytometry assay is performed in included subjects and controls. Reduced or absent burst activity will lead to genetic testing in search of overt immunodeficiency or susceptibility variants. All VEO-IBD patients will have an immunological work-up in search of a primary immunodeficiency.

Expected Results We anticipate to include a number of 150 pediatric patients with IBD over 12 months from the three pediatric gastroenterology units in Bucharest, Romania. We expect to identify an overall diminished neutrophil function in IBD patients versus controls and possible variants in the NADPH-complex genes.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: February 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

Subjects:

1. age under 18 years;

2. confirmed diagnosis of IBD fulfilling standard diagnosis criteria (clinical, radiological, endoscopical, histological features)

3. parental/guardian consent for study enrollment

Controls:

Healthy age-gender matched controls

Exclusion Criteria:

1. IBD associated to an already defined monogenic defect

2. Diagnosis of IBD uncertain

3. HIV/TB positive

Related Conditions & MeSH terms

• Chronic Granulomatous Disease
• Granulomatous Disease, Chronic
• Inflammatory Bowel Diseases
• Intestinal Diseases

Intervention

Diagnostic Test:
• Phagoburst
The main objective is to perform oxidative burst activity in pediatric patients with a definitive diagnosis of IBD and compare them with healthy controls.
• Lymphocytes subsets
To determine lymphocytes subsets in patients with suspicion of IEI
• Genetic testing
Perform targeted genetic testing in those with reduced to absent oxidative activity in search of PIDs and susceptibility variants

Locations

Country	Name	City	State
Romania	National Institute for Mother and Child Health Alessandrescu Rusescu	Bucharest	County

Sponsors (3)

Lead Sponsor	Collaborator
National Institute for Mother and Child Health Alessandrescu Rusescu	Marie Curie Emergency Children's Hospital, Bucharest, Victor Gomoiu Children's Hospital, Bucharest

Country where clinical trial is conducted

Romania, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Oxidative burst activity in IBD patients	Perform oxidative burst activity in pediatric patients with a definitive diagnosis of IBD and compare them with healthy controls.	12 months
Primary	Genetic testing in patients with suspicion of IEI	Perform targeted genetic testing in those with reduced to absent oxidative activity in search of PIDs and susceptibility variants	12 months
Secondary	Role of burst activity in disease phenotype	Find correlations between burst activity and disease activity	12 months