Clinical trial NCT01951833: Long Term Significance (Survival) of LCI in Patients With Cystic Fibrosis
Official title: Does Lung Clearance Index (LCI) Predict the Future Lung Disease Course in Patients With Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Cystic Fibrosis (CF)
Keywords: Lung clearance index (LCI)
Lead sponsor: University Hospital St Luc, Brussels
Sponsor class: OTHER
Collaborators: name: KU Leuven
First posted: 2013-09-27
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Cystic Fibrosis ( 6 years and older and FEV1 above 40% predicted) will be invited to participate in this prospective non interventional study in 2 CF centers (UCL and UZ Leuven)

Brief summary: The current dream in CF research is to discover safe drugs that correct the basic defect and prevent lung disease, allowing patients without significant lung damage to live nearly normal lives with a dramatic increase in life expectancy and without the burden of current treatment. The compound VX-770 (Ivacaftor Ò) is hoped to be the first milestone along this way. Progression of lung disease is now so gradual in many centres that sensitive indicators of early lung disease (small airways disease) are critically needed to assess the effects of such new treatments. In this context, assessment of ventilation inhomogeneity by the measurement called Lung clearance index (LCI) seems to be the most promising tool. However, to get approval by health authorities, new measures used in drug evaluation need to fulfill strict criteria. For LCI, the investigators still need to prove its long term significance: How well does the LCI measurement predict the long term lung disease course? Therefore, in this study the investigators want to measure LCI at baseline in a large patient cohort and establish how well it predicts the patients' disease course over the next 2 years.

Detailed description: Patients with CF ( 6 years and older and FEV1 above 40% predicted) will be invited to participate in this prospective non interventional study in 2 CF centers ( UCL and UZ Leuven). Jointly we take care of more than 40 % of the Belgian CF population. Median FEV1 of children (6 - \<18 y, n \>120) from these CTN centers is high ( around 100% pred), defining a study group of great interest in the current context.

To assess the predictive value of LCI over 2 years, the time to next pulmonary exacerbation will be evaluated in the entire cohort. To assess the predictive value of LCI over 2 years, the time to next pulmonary exacerbation will be evaluated in the entire cohort.

Repeat LCI measurements will be done at subsequent patient visits.

2 devices measuring the LCI will be tested and compared : Ecomedics and NDD.

LCI and spirometry will be measured in 120 healthy Belgian children and young adults. These subjects must be free of respiratory symptoms for at least two weeks and will not have any chronic or recurrent chest problem.

Eligibility criteria: Inclusion Criteria:

* FEV1(% predicted) \> 40%

Exclusion Criteria:

* FEV1(% predicted) \< 40%
* Exacerbation during baseline measurement

Primary outcomes:
measure: Lung Clearance Index (Predictive value and change of)
timeFrame: Baseline, 1 week, 3 months and 2 years
Secondary outcomes:
measure: FEV1 (Predictive value and change of)
timeFrame: baseline and 2 years
Other outcomes:
measure: Symptom score
timeFrame: 1 week, 3 months
Locations:
city: Woluwé-Saint-Lambert
country: Belgium
facility: Cliniques Universitaires Saint-Luc (UCL)
state: Bruxelles
Study officials:
affiliation: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
name: Lebecque Patrick, PhD
role: STUDY_DIRECTOR
affiliation: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
name: Poncin William
role: PRINCIPAL_INVESTIGATOR
References: