Risk Factors of Neonatal Respiratory Distress for Newborns With Prenatally Diagnosed Congenital Lung Malformations (MALFPULM)

October 31, 2022 updated by: Assistance Publique - Hôpitaux de Paris

Prospective Identification of Predictors of Neonatal Respiratory Distress for Newborns With Prenatally Diagnosed Congenital Lung Malformations : A Population-based, Nationally Representative Study

This research focuses on lung malformations detected in fetuses during prenatal ultrasound exams. Pathogenic mechanisms of these rare malformations are poorly understood. Improved knowledge is needed, to give families better information, and to better standardize treatment decisions The main goal is to better predict neonatal complications associated with these malformations, by identifying key predictive markers during the fetal period.

To achieve this objective, it is planned to include 400 pregnant women with prenatal diagnosis of pulmonary malformation in 45 health centers in France. This is the largest study on this topic at the international level.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The main objective of the study is to develop a prognostic model for estimating the risk of neonatal respiratory distress in children with prenatally diagnosed congenital pulmonary malformation.

The study will be offered to all pregnant women referred to a Center for Prenatal Diagnosis (CPD), due to the identification of a congenital lung malformations in the fetus. This study does not induce any changes in clinical and therapeutic monitoring proposed by the team in charge of the mother. At inclusion, and at each prenatal evaluation, prenatal parameters are entered in an e-CRF. In an effort to minimize any potential intra- and interoperator variability in malformation measurements over time, this study includes a standardized and centralized evaluation of ultrasound and MRI (if available) acquisitions of volume measurements. When the place of delivery is determined, a contact is made before birth with the teams (maternity, neonatology, intensive care unit), so that neonatal data are also collected prospectively. A phone call to the family is planned for the end of the first postnatal month, to identify any respiratory event that would have occurred between returning home after childbirth and the first month.

The routine follow-up of these children is then ensured in accordance with current national recommendations, in conjunction with the reference centers for rare respiratory diseases in children (28 university hospitals, spread across all regions of France). A telephone survey every 6 months with the referring physician in this specialized center or, alternatively, with the family, will collect clinical outcome until the age of 2 years. If a surgical intervention is planned within this interval, consent to collect part of the surgical specimen for research purposes will be solicited. This tissue will be immediately frozen at -80 ° C, to allow laser microdissection and DNA extraction from epithelial cells lining the malformation (Inserm U955). Frozen tissue will be conserved at the biobank of Necker-Enfants Malades.

Study Type

Observational

Enrollment (Actual)

436

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75015
        • Hôpital Necker - Enfants Malades

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Female

Sampling Method

Non-Probability Sample

Study Population

Pregnant women referred to a Center of Prenatal Diagnosis, because of the identification of a pulmonary malformation in the fetus

Description

Inclusion Criteria:

  • Prenatal identification of a congenital pulmonary malformation (hyperechoic and/or cystic pulmonary lesion)
  • consent of the mother for participation to the study

Exclusion Criteria:

- Absence of consent for participation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
identification of a pulmonary malformation in the fetus
pregnant women referred to a prenatal Center, because of the identification of a pulmonary malformation in the fetus
Other: identification of a pulmonary malformation in the fetus

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Respiratory distress
Time Frame: At Birth of the child
Respiratory distress at birth is defined by a breathing frequency > 60/min, or by the presence of chest retraction signs (Silverman score greater than or equal to 2). At least one of these signs must be persistent at 15' of life
At Birth of the child

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Necessity of antenatal treatment
Time Frame: At Birth of the child
Thoracic drainage, amniotic drainage, corticosteroids
At Birth of the child
Therapeutic abortion - fetal death
Time Frame: At Birth of the child
At Birth of the child
Severe respiratory distress
Time Frame: At Birth of the child
Severe respiratory distress at birth will be defined by the presence of at least one of the following parameters: persistent need at 15' of supplemental oxygen; Persistent need at 15' for a ventilatory support (non-invasive or invasive); neonatal death
At Birth of the child
Identification of KRAS mutation
Time Frame: 2 years
PCR analysis of known K-RAS mutations in codons 12 and 13
2 years
Level in delta Forskoline/IBMX Short Circuit Current (µA/cm2)
Time Frame: 2 years
CFTR activity evaluation
2 years
CFTR gene expression
Time Frame: 2 years
quantitative PCR
2 years
CFTR protein expression
Time Frame: 2 years
immunohistochemistry
2 years
Basal short circuit current : Isc Basal
Time Frame: 2 years
2 years
Effects of other potentiators on CFTR activity : ΔGenistein, ΔVX-770
Time Frame: 2 years
2 years
Inhibition of CFTR (inh-172) : ΔInh-172
Time Frame: 2 years
2 years
Response to ENaC inhibitors : ΔAmiloride, Δbenzamil
Time Frame: 2 years
2 years
Activation of Calcium Dependant Channels : ΔUTP
Time Frame: 2 years
2 years
Inhibition of SLC26A9 : ΔGlyH-101
Time Frame: 2 years
2 years
Response to inhibitors of basolateral K+ secretion : ΔBarium ; ΔChromanol
Time Frame: 2 years
2 years
Secretion of HCO3- in response to forskoline : Δ HCO3- primary culture
Time Frame: 2 years
2 years
Gene expression of other channels : ENaC, SLC26A9, CaCC, KVLQT1 and KCa3.1
Time Frame: 2 years
quantitative PCR
2 years
Protein expression of other channels : ENaC, SLC26A9, CaCC, KVLQT1 and KCa3.1
Time Frame: 2 years
immunohistochemistry
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Study Director: Laurent SALOMON, MD, PhD, Hospital Necker - Enfants Malades

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 17, 2015

Primary Completion (Actual)

November 30, 2018

Study Completion (Actual)

March 10, 2021

Study Registration Dates

First Submitted

January 5, 2015

First Submitted That Met QC Criteria

January 27, 2015

First Posted (Estimate)

February 2, 2015

Study Record Updates

Last Update Posted (Actual)

November 2, 2022

Last Update Submitted That Met QC Criteria

October 31, 2022

Last Verified

October 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NI13005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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