Changes in the respiratory microbial and biochemical environment after the start of CFTR targeted treatment in patients with cystic fibrosis

Completed

• Conditions: Cystic fibrosis; 10006436

• Registration Number: NL-OMON45428
• Lead Sponsor: Academisch Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 20

Inclusion Criteria

Patients with a (predominant) class II mutation (almost exclusively homozygous Phe508del) for cystic fibrosis (N<=20) who will be started on Ivacaftor/Lumacaftor (CFTR targeted therapy; Orkambi) therapy will be included in this longitudinal observational study.

Exclusion Criteria

For the bronchoscopy (part of the research protocol) the following patients will be excluded:
* Pre-lung transplant trajectory
* No informed consent for the procedure
* Deemed inappropriate by the treating physician.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
<p>* Bacterial sequences in sputum, broncho-alveolar lavage fluid and oral and<br /><br>nasal wash.<br /><br>* Metabolic profiles in broncho-alveolar lavage fluid and oral wash by mass<br /><br>spectrometry.<br /><br>* Volatile metabolites in breath. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>* Bacterial culture result from sputum and broncho-alveolar lavage fluid.<br /><br>* Lung function test results (FEV1/FVC, etc).<br /><br>* Routine plasma parameters of (chronic) inflammation (IgG, CRP, leukocyte<br /><br>count) and inflammatory profiles of sputum and broncho-alveolar lavage fluid<br /><br>measured by luminex.<br /><br>* Quality of life and fatigue assessments. </p><br>