Our primary objective is to determine the prognostic value of parameters for early disease detection of familial pulmonary fibrosis in the study population. All putative parameters for detection of preclinical disease have been shown to be…
ID
Source
Brief title
Condition
- Respiratory disorders congenital
- Lower respiratory tract disorders (excl obstruction and infection)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Presence of Interstitial Lung Disease (ILD) changes on HRCT are indicative of
preclinical interstitial lung disease and will be determined at baseline.
Putative parameters of early lung disease that will be investigated include
lung function, exercise tests, blood based biomarkers, MUC5B rs35705950
genotype, physical examination for digital clubbing and crackles, and
self-reported cough and dyspnoea.
The primary endpoint is the difference in these parameters between the group
with ILD changes on HRCT as compared to the group without ILD changes on HRCT.
Secondary outcome
The secondary endpoints are differences in values for parameters between the
baseline and follow-up screening after one and two years for the ILD changes
and no ILD changes cohort and differences between these groups.
Background summary
Familial pulmonary fibrosis (FPF) is a fatal lung disease that is often not
diagnosed until a significant portion of the lung function is lost. Median
survival after diagnosis is 3 to 5 years. As treatment can only slow down lung
function decline, early disease detection is essential to provide timely
therapeutic support. As first-degree relatives of patients with FPF are at high
risk of developing pulmonary fibrosis as well, a screening protocol has been
put in place. However, the value of current screening parameters to detect
early asymptomatic disease as well as the optimal interval between screening
appointments are unknown. A prospective study into the prognostic value of
these screening markers in the target population and the appropriate clinical
setting is needed to develop an evidence-based screening protocol. There will
be an emphasis on easily operable parameters that may allow for redirection of
(part of the) screening activities to the general practice in the future.
Study objective
Our primary objective is to determine the prognostic value of parameters for
early disease detection of familial pulmonary fibrosis in the study population.
All putative parameters for detection of preclinical disease have been shown to
be potentially informative due to their association with disease severity in
pulmonary fibrosis and observed presence in patients with preclinical disease.
As a secondary objective we aim to gain insight in the natural history of early
pulmonary fibrosis, as this is currently incompletely understood. In addition
we aim to determine the necessary interval between screening visits.
Study design
The study will be a prospective cohort study whereby subjects will visit the
hospital for screening at three time points: at baseline, after 1 year and
after 2 years.
Study burden and risks
Participants will visit the St Antonius Hospital 3 times in two years, visits
will take approximately 2:20 hour for the baseline visit and 1:50 hour for the
two subsequent visits. The health-related burden for participants is minimal
and consists of physical examination (including auscultation with electronic
stethoscope), three times blood withdrawal, 6-minute walking test and pulmonary
function testing and one HRCT scan. Except for the 6-minute walking test and
the auscultation with the electronic stethoscope, these procedures would
otherwise also be performed as part of the routine lung screening for pulmonary
fibrosis in the context of familial disease. Measures are taken to facilitate
the practical burden of visiting and costs.
Koekoekslaan 1
Nieuwegein 3435 CM
NL
Koekoekslaan 1
Nieuwegein 3435 CM
NL
Listed location countries
Age
Inclusion criteria
Subjects must be a first-degree relative of a patient with familial pulmonary
fibrosis
Subjects are reffered by a medical doctor for screening of familial pulmonary
fibrosis
Exclusion criteria
- A previous diagnosis of an interstitial lung disease (ILD)
- Pregnancy
Design
Recruitment
Medical products/devices used
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 |
|---|---|
| ClinicalTrials.gov | NCT05367349 |
| CCMO | NL75303.100.20 |