Respiratory Function at Preschool Age of Children Detected of Cystic Fibrosis in Neonatal Period

Not Applicable

Completed

• Conditions: Cystic Fibrosis
• Interventions: Other: 3 years-assessment of respiratory function

• Registration Number: NCT02358798
• Lead Sponsor: University Hospital, Montpellier

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-08-19
• Study First Submitted: 2015-01-21
• Study First Submitted QC: 2015-02-03
• Study First Posted: 2015-02-09
• Primary Completion: 2020-01-17
• Study Completion: 2020-01-17
• Status Verified: 2020-04
• Last Update Submitted: 2020-04-09
• Last Update Posted: 2020-04-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Preschool aged children detected of CF in neonatal period	3 years-assessment of respiratory function	Preschool aged children detected of Cystic Fibrosis in neonatal period

Primary Outcome Measures

Name	Time	Method
Time evolution of functional residual capacity by Helium dilution technique (CRF He)	at each four yearly routine visits

Secondary Outcome Measures

Name	Time	Method
plethysmographic measure of specific resistance (sRaw)	at each four yearly routine visits
pulmonary clearance index	at each four yearly routine visits
airway resistance by debit interruption technique (Rint)	at each four yearly routine visits
arterial blood gas measurement	at each four yearly routine visits
flow-volume curve	at each four yearly routine visits
measure of administration antibiotics and antiasthmatics treatments	at each four yearly routine visits	Comparison of the evolution of these parameters to changing those of a historical cohort evaluated before the introduction of neonatal screening.
measurement of organ damage	at each four yearly routine visits
measurement of tobacco exposition	at each four yearly routine visits

Trial Locations

Locations (1)

University Hospital of Montpellier, Arnaud de Villeneuve; 🇫🇷 Montpellier, France