Clinical trial NCT02358798: Respiratory Function at Preschool Age of Children Detected of Cystic Fibrosis in Neonatal Period
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Keywords: Time evolution of respiratory function
Longitudinal multicenter monitoring of large cohorts
Preschool aged children
Detection of cystic fibrosis in neonatal period
Lead sponsor: University Hospital, Montpellier
Sponsor class: OTHER
First posted: 2015-02-09
Enrollment: 40
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: The widespread neonatal detection of cystic fibrosis in France since 2002 permits to treat children from birth. New treatments used for young children involve to assess efficacy criteria specific to this population. Standard respiratory function criteria for older children and adults is forced expiratory volume/second.

This technique is not suited for preschool aged children (3 to 6 years old) because they are too old to be sedated and too young and immature to be able to make forced expiration technique that are correct, reproducible and prolonged during more than 1 second.

For preschool aged children, in order to assess distal damage and her consequence, the evaluations are: airway resistance by debit interruption technic (Rint), plethysmographic measure of specific resistance (sRaw), functional residual capacity by Helium dilution technique (CRF He), arterial blood gas measurement, pulmonary clearance index.

All these methods have a better success rate and can be used in alternative or with forced spirometry. However, each of them gives only a part of information on airway and lung damage of detected children. It is necessary to combine them for a better information on overall respiratory damage.

In France, each respiratory function test laboratory uses one or any of these methods in addition to flow-volume curve, in function of his practices and his equipment.

So, respiratory function test of preschool aged children is going to diversify more and more to the detriment of an homogeneity of practices between different centers.

A referent population during a longitudinal multicenter monitoring on large cohorts that describe the evolution of pulmonary function, obtained by a standardized methodology is necessary to assess the efficacy of any new treatment. And, with the homogenization of care of children detected of cystic fibrosis in different centers, the description of natural evolution of pulmonary function by a standardized methodology will improve the discriminative power of measure of respiratory function to assess the presence of a worsening in preschool-aged children.


Eligibility criteria: Inclusion Criteria:

* Patient suffering from cystic fibrosis
* Height between 90 et 130cm
* No respiratory exacerbation since 4 weeks
* Benefit from an insurance disease regime

Exclusion Criteria:

* Law-protected patient
* Patient's parent don't understand french language
* Opposition to participation

Primary outcomes:
measure: Time evolution of functional residual capacity by Helium dilution technique (CRF He)
timeFrame: at each four yearly routine visits
Secondary outcomes:
measure: airway resistance by debit interruption technique (Rint)
timeFrame: at each four yearly routine visits
measure: plethysmographic measure of specific resistance (sRaw)
timeFrame: at each four yearly routine visits
measure: arterial blood gas measurement
timeFrame: at each four yearly routine visits
measure: pulmonary clearance index
timeFrame: at each four yearly routine visits
measure: flow-volume curve
timeFrame: at each four yearly routine visits
measure: measurement of organ damage
timeFrame: at each four yearly routine visits
measure: measurement of tobacco exposition
timeFrame: at each four yearly routine visits
measure: measure of administration antibiotics and antiasthmatics treatments
timeFrame: at each four yearly routine visits
Other outcomes:
Locations:
city: Montpellier
country: France
facility: University Hospital of Montpellier, Arnaud de Villeneuve
Study officials:
affiliation: University Hospital, Montpellier
name: MATECKI SM Stephan, MD
role: PRINCIPAL_INVESTIGATOR
References: