Chronic Granulomatous DiseaseNCT06162936
Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease
An observational study of Chronic Granulomatous Disease, run by National Institute for Mother and Child Health Alessandrescu Rusescu at 1 site in Romania.
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. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- Phagoburst, Lymphocytes subsets and Genetic testing
- How many people
- 150 participants
- Where
- 1 siteRomania
- Study dates
- 1 Nov 2023to Feb 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The study population will include pediatric patients diagnosed with IBD (right from the diagnosis or anytime during the disease course) from three different gastroenterology units in Bucharest, Romania. Screening tests for an imunological disease (neutrophil oxidative burst, lymphocyte subsets) will be performed in included patients and healthy controls by the home institution.
Who may be able to join
- Subjects:
- age under 18 years;
- confirmed diagnosis of IBD fulfilling standard diagnosis criteria (clinical, radiological, endoscopical, histological features)
- parental/guardian consent for study enrollment
- Controls:
- Healthy age-gender matched controls
Who cannot join
- IBD associated to an already defined monogenic defect
- Diagnosis of IBD uncertain
- HIV/TB positive
Trial locations and contacts
1 site in 1 country.
Romania1 site, 1 recruiting
- Recruiting
National Institute for Mother and Child Health Alessandrescu Rusescu
Bucharest, County, 041249
Alexis Virgil Cochino, MD, PhD, +40723196648, alexis_virgil@yahoo.com
I
About this study
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.
Read more
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.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- Phagoburst, Lymphocytes subsets, Genetic testing
- Keywords
- inflammatory bowel disease, chronic granulomatous disease, inborn errors of immunity
- Medical subject headings
- Granulomatous Disease, Chronic, Inflammatory Bowel Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PhagoburstDiagnostic test
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 subsetsDiagnostic test
To determine lymphocytes subsets in patients with suspicion of IEI
Genetic testingDiagnostic test
Perform targeted genetic testing in those with reduced to absent oxidative activity in search of PIDs and susceptibility variants
| Study group | Role | What they receive |
|---|---|---|
| Inflammatory bowel disease patients | Pediatric patients (under 18 years of age) diagnosed with IBD Receives: Diagnostic Test: Phagoburst; Diagnostic Test: Lymphocytes subsets; Diagnostic Test: Genetic testing | |
| Healthy controls | Healthy chidren (no chronic disease) Receives: Diagnostic Test: Phagoburst |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months
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
Measured: 12 months
Secondary outcomes (1)
Role of burst activity in disease phenotype
Find correlations between burst activity and disease activity
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2023
- Study start1 Nov 2023
- Primary completionDec 2024 (estimated)
- Study completionFeb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Institute for Mother and Child Health Alessandrescu Rusescu
- Sponsor type
- University, hospital or other
- Collaborators
- Marie Curie Emergency Children's Hospital, Bucharest, Victor Gomoiu Children's Hospital, Bucharest
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06162936
- Sponsor's protocol ID
- IBD20232024
- Status last verified
- Nov 2023
- First submitted
- 30 Nov 2023
- Last updated
- 8 Dec 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neutrophil Oxidative Burst in Early and Late Onset Pediatric Inflammatory Bowel Disease
- ClinicalTrials.gov ID
- NCT06162936
- Acronym / sponsor ID
- IBD20232024
- Conditions
- Chronic Granulomatous Disease
- Keywords
- inflammatory bowel disease, chronic granulomatous disease, inborn errors of immunity
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- National Institute for Mother and Child Health Alessandrescu RusescuUniversity, hospital or other
- Collaborators
- Marie Curie Emergency Children's Hospital, Bucharest, Victor Gomoiu Children's Hospital, Bucharest
- Study start
- 1 Nov 2023 (actual)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Feb 2025 (estimated)
- First posted
- 8 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Inflammatory bowel disease patients | – | Diagnostic Test: Phagoburst, Diagnostic Test: Lymphocytes subsets, Diagnostic Test: Genetic testingPediatric patients (under 18 years of age) diagnosed with IBD |
| Healthy controls | – | Diagnostic Test: PhagoburstHealthy chidren (no chronic disease) |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study population will include pediatric patients diagnosed with IBD (right from the diagnosis or anytime during the disease course) from three different gastroenterology units in Bucharest, Romania. Screening tests for an imunological disease (neutrophil oxidative burst, lymphocyte subsets) will be performed in included patients and healthy controls by the home institution.
Key inclusion criteria (6 listed)
- age under 18 years;
- confirmed diagnosis of IBD fulfilling standard diagnosis criteria (clinical, radiological, endoscopical, histological features)
- parental/guardian consent for study enrollment
- Healthy age-gender matched controls
Key exclusion criteria (3 listed)
- IBD associated to an already defined monogenic defect
- Diagnosis of IBD uncertain
- HIV/TB positive
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PhagoburstThe main objective is to perform oxidative burst activity in pediatric patients with a definitive diagnosis of IBD and compare them with healthy controls. | Diagnostic test | Healthy controls, Inflammatory bowel disease patients |
| Lymphocytes subsetsTo determine lymphocytes subsets in patients with suspicion of IEI | Diagnostic test | Inflammatory bowel disease patients |
| Genetic testingPerform targeted genetic testing in those with reduced to absent oxidative activity in search of PIDs and susceptibility variants | Diagnostic test | Inflammatory bowel disease patients |
Primary outcome
Oxidative burst activity in IBD patients
Time frame: 12 months
Perform oxidative burst activity in pediatric patients with a definitive diagnosis of IBD and compare them with healthy controls.
Genetic testing in patients with suspicion of IEI
Time frame: 12 months
Perform targeted genetic testing in those with reduced to absent oxidative activity in search of PIDs and susceptibility variants
1 secondary outcome
Role of burst activity in disease phenotype
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Chronic Granulomatous Disease, run by National Institute for Mother and Child Health Alessandrescu Rusescu at 1 site in Romania.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Romania. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alexis Virgil Cochino, MD, PhD (+40723193648, alexis_virgil@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Oxidative burst activity in IBD patients (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2023.