Inflammatory Markers in Broncho-alveolar Lavage Fluid as Risk Factors for Lung Disease in Infants With Cystic Fibrosis: the I-BALL Study

NCT02907788 | Completed DMC

• 1 other identifier: NL49725.078.14
• Study Type: observational
• Target: 51
• Locations: 1 country
• Sites: 1

Brief Summary

Airway disease, featuring intense inflammation, is the main cause of morbidity and mortality in cystic fibrosis (CF). Mechanisms of CF airway inflammation remain unclear, hampering development of better treatments.This time-sensitive ancillary study leverages a unique longitudinal cohort of CF infants, assessing the early phase of airway disease. Through the use of innovative cell and fluid based tools for in vivo profiling and in vitro testing of BALF samples, this translational effort will yield unprecedented insights into mechanisms of PMN dysfunction in CF, and assess new paths for early intervention.

Conditions

Cystic Fibrosis

Keywords

Cystic fibrosis; Neutrophils; PMN; Inflammation; Lung disease; PRAGMA

Outcome Measures

Primary Outcomes (2)

• Lipid profiles with early lung disease in CF.

  Lipidomics endpoints: The primary end-points are the different bioactive lipid levels in BALF of infants with CF using liquid chromatography (LC) coupled to Mass spectrometry (MS). Lipid profiles will be derived of the BALF supernatant

  5 years

• Surface markers of reprogrammed PMNs in BALF of infants with CF

  Our primary endpoint for BALF cells flowcytometry analysis is surfacemarkers on airway PMNs (exocytosis of NE-rich granules), which was shown to correlate with lung function in chronic CF disease

  5 years

Secondary Outcomes (1)

• PRAGMA-CT scores of infants with CF

  5 years

Study Arms (2)

Cystic Fibrosis patients

Patient with cystic fibrosis diagnosed by Heel-prick screening

non-CF patients

Children who undergo bronchoscopy for another reason, without CF: eg gastro-esophageal reflux.

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

All CF patients enrolled in the AREST-CF program of the paediatric CF centre at Sophia Children's Hospital and Wilhelmina Children's Hospital.

You may qualify if:

• Diagnosed with CF, confirmed with 2 mutations found by genetic analysis, either from heel-prick screening or diagnosed later in life
• Aged 3 months (Utrecht),1, 3 or 5 years, who undergo bronchoscopy and chest CT scan as part of the routine monitoring program for CF
• Informed consent from parents

You may not qualify if:

• Absence of previously given informed consent for use of encoded clinical data for scientific purposes

Sponsors & Collaborators

• Erasmus Medical Center: lead
• Emory University: collaborator
• UMC Utrecht: collaborator

Study Sites (1)

Erasmus MC -Sophia childrens hospital

Rotterdam, South Holland, 3015 CN, Netherlands

Location

Biospecimen

Retention: SAMPLES WITH DNA

* Whole Blood * Bronchoscopies and bronchoalveolar lavage fluid (BALF)

MeSH Terms

Conditions

Cystic Fibrosis; Inflammation; Lung Diseases

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Rabindra Tirouvanziam, Assistant Professor

  Emory University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dr, MD, PhD

Study Record Dates

• First Submitted: September 6, 2016
• First Posted: September 20, 2016
• Study Start: September 1, 2014
• Primary Completion: January 1, 2021
• Study Completion: January 1, 2021
• Last Updated: November 5, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)