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

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.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Other: 3 years-assessment of respiratory function

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Montpellier, France, 34295
    • University Hospital of Montpellier, Arnaud de Villeneuve

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Preschool aged children detected of CF in neonatal period; Preschool aged children detected of Cystic Fibrosis in neonatal period	Other: 3 years-assessment of respiratory function

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University Hospital, Montpellier
• Investigators: Principal Investigator: MATECKI SM Stephan, MD, University Hospital, Montpellier

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2014
• Primary Completion (Actual): January 17, 2020
• Study Completion (Actual): January 17, 2020

Study Registration Dates

• First Submitted: January 21, 2015
• First Submitted That Met QC Criteria: February 3, 2015
• First Posted (Estimate): February 9, 2015

Study Record Updates

• Last Update Posted (Actual): April 14, 2020
• Last Update Submitted That Met QC Criteria: April 9, 2020
• Last Verified: April 1, 2020

More Information

Terms related to this study

• Keywords: Preschool aged children; Time evolution of respiratory function; Longitudinal multicenter monitoring of large cohorts; Detection of cystic fibrosis in neonatal period
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 9393