Impact of CFTR-modulation with elexacaftor/tezacaftor/ivacaftor on the exercise capacity, intestinal and respiratory microbiome, microbial and inflammatory metabolites in cystic fibrosis

• Conditions: E84.0; Cystic fibrosis with pulmonary manifestations

• Registration Number: DRKS00023862
• Lead Sponsor: Zentrum für Kinder- und Jugendmedizin der Universitätsmedizin Mainz

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-06-25
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Complete
• Sex: All
• Target Recruitment: 84

Inclusion Criteria

Age 6 and older
- Confirmed diagnosis and mutation
- Planned to start medication with elexacaftor/tezacaftor/ivacaftor
- Written informed consent (from parent or legal guardian if underaged)

Or sex- and aged matched healthy control

Exclusion Criteria

- Comorbidity which exclude therapy with elexacaftor/tezacaftor/ivacaftor (e.g liver or renal insufficiency)
- Respiratory infection at the start of treatment
- Acute therapy changes 21 days before starting therapy with elexacaftor/tezacaftor/ivacaftor
- Antibiotic treatment up to 3 weeks prior to study start

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ongitudinal changes in diversity, species richness and in microbial composition of respiratory and intestinal microbiome and in exercise capacity (peak oxygen uptake and maximal work rate) during the therapy with CFTR-modulators.

Secondary Outcome Measures

Name	Time	Method
Secondary objectives include<br>- Evaluation of systemic inflammatory parameter (CRP, IgG, IL-8, TNF-alpha, TGF-beta), intestinal and airway inflammation (faecal calprotectin, Il-8 and NE in sputum) and urine cathepsine.<br>- Lung function measurement with spirometry and MBW will be performed to assess absolute changes in ppFEV1, ppFVC, ppFEV75-25 and in LCI.<br>- Changes in concentration of sweat chloride.<br>- Cystic Fibrosis Questionnaire-Revised (CFQ-R) will be completed at all visits.<br>- Assessment of CF metabolome by mass spectroscopy for CF-specific biomarkers.