I-BALL 2: Non-invasive Disease Monitoring in Infants with Cystic Fibrosis

Completed

• Conditions: CF; Cystic Fibrosis; 10010613; 10038686; 10004018

• Registration Number: NL-OMON49034
• Lead Sponsor: Erasmus MC, Universitair Medisch Centrum Rotterdam

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 120

Inclusion Criteria

Subjects are eligible for this study when they are:
• Diagnosed with CF, either by abnormal sweat test and/or confirmed with 2
mutations found by genetic analysis, either from heel-prick screening or
diagnosed later in life.
• Aged 1, 3 or 5 years, when they undergo bronchoscopy and chest CT scan as
part of the routine monitoring program for CF.
• Aged 1, 2, 3, 4, or 5 years, when they visit the outpatient clinic for
routine yearly follow-up, the patient will be eligible for induced Sputum,
exhaled breath collection and blood collection for TiMaSCAN.
• Authorized by a written informed consent from parents to undergo a sputum
induction to collect Induced Sputum (IS), to collect exhaled breath for
Volatile Organic Compounds (VOC) analysis, to collect an extra vial of blood
during the routine venous puncture, a nasopharyngeal swab (during anesthesia
when applicable) and permission to use excess biomaterials, especially BALF
retrieved during bronchoscopy and coded clinical data for research. Parents
may choose to opt in or out for separate parts of the study.

Exclusion Criteria

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

Study & Design

• Study Type: Observational non invasive
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>1. Inflammatory markers in BAL, validated against CT score and LCI<br /><br>2. Inflammatory markers in IS and EB, validated against BAL, CT and LCI.<br /><br>3. The detection of intracellular pathogens in peripheral blood monocytes using<br /><br>TiMaSCAN, validated </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Chest CT scores according to the PRAGMA scoring system<br /><br>• Lung Clearance Index as a functional endpoint<br /><br>• Parent reported symptoms will be recorded with the use of a questionnaire.<br /><br>• TiMaSCAN secondary endpoints<br /><br>o Assess agreement between TiMaSCAN results and reported<br /><br>clinical symptoms.<br /><br>o We will determine the prevalence of the different<br /><br>microorganisms that are detected using TiMaSCAN within infected individuals,<br /><br>i.e. those with a positive TiMaSCAN result.<br /><br>This can be determined irrespective of the presence of symptoms. Data on the<br /><br>prevalence of specific pathogens generated<br /><br>using TiMaSCAN will be compared with the results derived from airway cultures. </p><br>