Exploratory Observational Prospective Study in Neonatal and Pediatric Congenital Diaphragmatic Hernia

NCT ID: NCT04114578

Last Updated: 2024-11-22

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 80 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-10-01
• Study Completion Date: 2025-06-30

Brief Summary

CDH is associated with lung hypoplasia, pulmonary hypertension, and left ventricular hypoplasia.

Use of new STE techniques (heart ultrasound) showed that CDH newborns have decreased LV size and function, potentially explaining the non-response to iNO, and that these cardiac findings were associated with poor outcomes. Our hypothesis: CDH newborns persist to have some degree of LV hypoplasia in the pediatric and adolescent life and pulmonary pressures remain increased during growth. Patients with decreased cardiac performance by STE and/or with PH have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile

Detailed Description

The principal investigator hypothesize that CDH newborns have disturbed cardiac performance and increased pulmonary pressures during neonatal life and at different pediatric ages. The principal investigator also postulate that those with decreased cardiac performance by STE and/or with PH have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile, that CDH patients have some remaining degree of LV hypoplasia at pediatric/adolescent age and that novel echocardiography techniques will detect anomalies not uncovered by conventional imaging.

This study is a prospective study. Cohorts will be simultaneously recruited at different neonatal and pediatric ages to create various age groups of the CDH populations.

Groups will be synchronized with the current follow-up programs to ease feasibility.

Patient Population will be CDH patients admitted to the NICU at the MCH or followed in the clinic. Patients will be recruited during hospitalization or at the different age groups corresponding to their regular CDH follow-up.

Echocardiography will be targeted to acquire: RV and LV function by STE, 3D derived RV/LV volumes and performance , conventional systolic/diastolic RV and LV function parameters (such as: ejection fraction by Simpson's Biplane, tricuspid annular plane systolic excursion and tissue Doppler velocities), cardiac outputs and pulmonary pressure estimates. For patient's safety, responsible clinician will be alerted when a clinically significant anomaly is detected on the research echocardiography. Same echocardiography protocol will be applied to controls.

Associated to echocardiography data, The principal investigator will complete a chart review to extract important neonatal/pediatric demographic and clinical data. Variables extracted, among others, will include perinatal details (mode of delivery, maternal conditions, APGAR scores), sex, birth weight, clinical course and major morbidities. Bayley Scales of Infant and Toddler Development - Third Edition, a standardized evaluation for motor, cognitive and language abilities, is done on all patients with CDH at 18 months PMA at the MCH. When available for age groups beyond 18 months of age, these scores will be recorded. As well, assessment tools describing functional and developmental status will be filled with parents (or legal guardian) and/or pre-adolescents/adolescents. These tools will be the: Ages & Stages Questionnaires 3 (a parent report of developmental skills for ages of 2 to 60 months , the Clinical Adaptive Test /Clinical Linguistic Auditory Milestone Scale (CAT/CLAMS), which has a high correlation with the Bayley Scales of Infant Development and is a neurodevelopmental assessment tool of infants and toddlers) and the Pediatric Quality of Life Inventory Survey (a pre-adolescent and adolescent report on quality of life and functional status).

Patients with CDH will be compared to controls (1:1) recruited contemporaneously. Clinics targeted for control groups include the dermatology, orthopedic, trauma follow-up and neurology clinic for benign seizures episodes.

Conditions

Congenital Diaphragmatic Hernia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Neonatal profile

ECHO in first 24 hours of life - ideally ECHO at Day 3-5 of life ECHO at 2-3 weeks of life or before discharge (whichever comes first) Data collected at each echocardiography: blood pressure at beginning of ECHO, pre and post-ductal saturation, respiratory support, use of inotropes and dosages, use of iNO and dosage and last blood gas

Echocardiography

Intervention Type: OTHER

Echocardiography has become routinely used in the diagnosis, management, and follow-up of patients with any suspected or known heart diseases. It is one of the most widely used diagnostic tests in cardiology. It can provide a wealth of helpful information, including the size and shape of the heart (internal chamber size quantification), pumping capacity, and the location and extent of any tissue damage. An echocardiogram can also give physicians other estimates of heart function, such as a calculation of the cardiac output, ejection fraction, and diastolic function (how well the heart relaxes). The acquisition usually takes about 30 minutes.

Infant profile ( 4 month and/or 9 month)

Echocardiography Age and stage questionnaires CAT/CLAMS assessment

Echocardiography

Intervention Type: OTHER

Echocardiography has become routinely used in the diagnosis, management, and follow-up of patients with any suspected or known heart diseases. It is one of the most widely used diagnostic tests in cardiology. It can provide a wealth of helpful information, including the size and shape of the heart (internal chamber size quantification), pumping capacity, and the location and extent of any tissue damage. An echocardiogram can also give physicians other estimates of heart function, such as a calculation of the cardiac output, ejection fraction, and diastolic function (how well the heart relaxes). The acquisition usually takes about 30 minutes.

Pediatric profile 3, 5 and/or 8years

Echocardiography Age and stage questionnaires CAT/CLAMS assessment Results from 18 months PMA Bailey will be retrieved

Echocardiography

Intervention Type: OTHER

Echocardiography has become routinely used in the diagnosis, management, and follow-up of patients with any suspected or known heart diseases. It is one of the most widely used diagnostic tests in cardiology. It can provide a wealth of helpful information, including the size and shape of the heart (internal chamber size quantification), pumping capacity, and the location and extent of any tissue damage. An echocardiogram can also give physicians other estimates of heart function, such as a calculation of the cardiac output, ejection fraction, and diastolic function (how well the heart relaxes). The acquisition usually takes about 30 minutes.

Pre-adolescent/adolescent profile 11,14 and/or 17 years

Echocardiography Pediatric Quality of Life inventory survey

Echocardiography

Intervention Type: OTHER

Echocardiography has become routinely used in the diagnosis, management, and follow-up of patients with any suspected or known heart diseases. It is one of the most widely used diagnostic tests in cardiology. It can provide a wealth of helpful information, including the size and shape of the heart (internal chamber size quantification), pumping capacity, and the location and extent of any tissue damage. An echocardiogram can also give physicians other estimates of heart function, such as a calculation of the cardiac output, ejection fraction, and diastolic function (how well the heart relaxes). The acquisition usually takes about 30 minutes.

Interventions

Echocardiography

Echocardiography has become routinely used in the diagnosis, management, and follow-up of patients with any suspected or known heart diseases. It is one of the most widely used diagnostic tests in cardiology. It can provide a wealth of helpful information, including the size and shape of the heart (internal chamber size quantification), pumping capacity, and the location and extent of any tissue damage. An echocardiogram can also give physicians other estimates of heart function, such as a calculation of the cardiac output, ejection fraction, and diastolic function (how well the heart relaxes). The acquisition usually takes about 30 minutes.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• CDH patients admitted to the MCH-NICU
• CDH followed at the MCH CDH clinic

Exclusion:

• Prematurity (< 36 weeks)
• Diagnosis at > 7 days of life
• Bilateral CDH
• Congenital cardiac defect (excluding a patent ductus arteriosus (PDA), atrial septal defect (ASD) or ventricular septal defect (VSD)) and/or other major anomaly/genetic syndrome.

• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

McGill University Health Centre/Research Institute of the McGill University Health Centre

OTHER

Sponsor Role: lead

Responsible Party

Gabriel Altit

Neonatologist

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Mcgill University Health Centre

Montreal, Quebec, Canada

Site Status: RECRUITING

Countries

Canada

Facility Contacts

Gabriel Altit, MD

Role: primary

gabriel.altit@mail.mcgill.ca

514-412-4452

Other Identifiers

2020-4892

Identifier Type: -

Identifier Source: org_study_id