We aim to evaluate the changes in the composition and the function of the respiratory microbiome after the initiation of targeted CFTR therapy. Second we want to relate the change in respiratory biochemical and microbial environment to clinical…
ID
Source
Brief title
Condition
- Bronchial disorders (excl neoplasms)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Four visits are planned per patient as part of standard care. Material will
obtained during all visits. Lung function, microbial cultures of sputum and
blood sampling will be performed as part of routine care. An oral wash, nasal
wash, and exhaled breath, and faeces and DBS with a finger prick collection
will be obtained as additional procedure. Functional metagenomics and
metabolomics analysis will be performed on sputum samples, oral and nasal wash,
and faeces samples. The primary endpoint is the change in bacterial diversity
after the start of Elexacaftor/Tezacaftor/Ivacaftor.
Secondary outcome
NA
Background summary
The lungs of patients with CF are characterized by (1) impaired mucus
clearance, (2) acidic milieu, (3) increased number of neutrophils and (4)
increased bacterial loads. Novel therapies target the CF transmembrane
conductance regulator (CFTR) and increase its activity. A novel triple CFTR
targeted therapy is shown to have a major effect on pulmonary function,
mucociliary clearance and reduction in sweat chloride test. However, very
little is known about the influence of the targeted CFTR therapies on the
respiratory microbiome. One of the major challenges in CF is to limit the
colonization of the respiratory tract by well-adapted microbes such as
Pseudomonas and Achromobacter and maintain a healthy respiratory flora.
Study objective
We aim to evaluate the changes in the composition and the function of the
respiratory microbiome after the initiation of targeted CFTR therapy. Second we
want to relate the change in respiratory biochemical and microbial environment
to clinical changes (for example lung function). Third we relate the changes of
the respiratory and gut microbiome. Fourth, we will explore the differences in
pre-medication respiratory microbiome / metabolome between patients that
clinically respond and do not respond to treatment. Finally, we will clinically
validate a dried blood spot (DBS) sampling method.
Study design
Longitudinal observational cohort study.
Study burden and risks
All assessment will be performed in conjunction with routine visits to the
outpatient clinic as much as possible. The most important addition procedure is
the exhaled breath sampling, and faeces and DBS collection, both non-invasive
procedures. The patient will not have benefit from participation in the study.
We aim for improved treatment of bacterial dysbalance in the respiratory tract
of all patients with CF and in that respect the results of the study may
improve treatment in the future for the patients participating in the study or
any patient with similar characteristics.
Meibergdreef 9
Amsterdam 1105AZ
NL
Meibergdreef 9
Amsterdam 1105AZ
NL
Listed location countries
Age
Inclusion criteria
Start with Elexa-/Teza-/Ivacaftor therapy on basis of compassionate use
Exclusion criteria
none
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| CCMO | NL70667.018.19 |