Congenital Diaphragmatic HerniaNCT04114578
Exploratory Observational Prospective Study in Neonatal and Pediatric Congenital Diaphragmatic Hernia
An observational study of Congenital Diaphragmatic Hernia, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 17 yearsHealthy volunteers can take part
- Conditions the study is for
- Congenital Diaphragmatic Hernia
- What is being tested?
- Echocardiography
- How many people
- 80 participants
- Where
- 1 siteCanada
- Study dates
- 1 Oct 2019to 30 Jun 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- Healthy volunteers accepted
- Child
Study population: The control groups will be recruited in the normal nursery, in the NICU (term infants with antenatal suspicion of coarctation, which is ruled-out postnatally) or in clinics with conditions non-related to lungs or heart (such as: Inguinal hernia, Dermatology, Orthopedic and Benign Chest Pain clinics), as described in our previous published studies .
Who may be able to join
- CDH patients admitted to the MCH-NICU
- CDH followed at the MCH CDH clinic
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Canada1 site, 1 recruiting
- Recruiting
Mcgill University Health Centre
Montreal, Quebec, H4A 3J1
Gabriel Altit, MD, 514-412-4452, gabriel.altit@mail.mcgill.ca
About this study
Official title: ECHO-CDH - Exploratory Observational Prospective Study in Neonatal and Pediatric Congenital Diaphragmatic Hernia
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
Read the 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
- Being tested
- Echocardiography
- Medical subject headings
- Hernias, Diaphragmatic, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EchocardiographyOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Other: Echocardiography | |
| Infant profile ( 4 month and/or 9 month) | Echocardiography Age and stage questionnaires CAT/CLAMS assessment Receives: Other: Echocardiography | |
| Pediatric profile 3, 5 and/or 8years | Echocardiography Age and stage questionnaires CAT/CLAMS assessment Results from 18 months PMA Bailey will be retrieved Receives: Other: Echocardiography | |
| Pre-adolescent/adolescent profile 11,14 and/or 17 years | Echocardiography Pediatric Quality of Life inventory survey Receives: Other: Echocardiography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Measured: Within 24 hours of life
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Measured: Between day 3 to 5 of life
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Measured: Between 2 to 3 weeks of life
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Measured: age of 4 and/or 9 months
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Measured: 3, 5 and/or 8 years
Assessment of cardiac function
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain) by %
Measured: 11, 14 and/or 17 years
Secondary outcomes (6)
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: Within 24 hours of life
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: Between day 3 to 5 of life
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: Between 2 to 3 weeks of life
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: age of 4 and/or 9 months
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: 3, 5 and/or 8 years
Prevalence of pulmonary hypertension
CDH newborns with pulmonary hypertension have higher concomitant neonatal or pediatric morbidities and altered neurodevelopmental profile (by mm/Hg)
Measured: 11, 14 and/or 17 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2019
- Study start1 Oct 2019
- Primary completion31 Dec 2023
- Study completion30 Jun 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Diaphragmatic HerniaStudies of the same condition, open and under way.
- More trials run by McGill University Health Centre/Research Institute of the McGill University Health CentreThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Gabriel Altit, McGill University Health Centre/Research Institute of the McGill University Health Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04114578
- Sponsor's protocol ID
- 2020-4892
- Status last verified
- Nov 2024
- First submitted
- 27 Sep 2019
- Last updated
- 22 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ECHO-CDH - Exploratory Observational Prospective Study in Neonatal and Pediatric Congenital Diaphragmatic Hernia
- ClinicalTrials.gov ID
- NCT04114578
- Acronym / sponsor ID
- 2020-4892
- Conditions
- Congenital Diaphragmatic Hernia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health CentreUniversity, hospital or other
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 30 Jun 2025 (estimated)
- First posted
- 3 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Neonatal profile | – | Other: EchocardiographyECHO 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 |
| Infant profile ( 4 month and/or 9 month) | – | Other: EchocardiographyEchocardiography Age and stage questionnaires CAT/CLAMS assessment |
| Pediatric profile 3, 5 and/or 8years | – | Other: EchocardiographyEchocardiography Age and stage questionnaires CAT/CLAMS assessment Results from 18 months PMA Bailey will be retrieved |
| Pre-adolescent/adolescent profile 11,14 and/or 17 years | – | Other: EchocardiographyEchocardiography Pediatric Quality of Life inventory survey |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
- Study population
- The control groups will be recruited in the normal nursery, in the NICU (term infants with antenatal suspicion of coarctation, which is ruled-out postnatally) or in clinics with conditions non-related to lungs or heart (such as: Inguinal hernia, Dermatology, Orthopedic and Benign Chest Pain clinics), as described in our previous published studies .
Key inclusion criteria (2 listed)
- CDH patients admitted to the MCH-NICU
- CDH followed at the MCH CDH clinic
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EchocardiographyEchocardiography 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. | Other | Infant profile ( 4 month and/or 9 month), Neonatal profile, Pediatric profile 3, 5 and/or 8years, Pre-adolescent/adolescent profile 11,14 and/or 17 years |
Primary outcome
Assessment of cardiac function
Time frame: Within 24 hours of life
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Assessment of cardiac function
Time frame: Between day 3 to 5 of life
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Assessment of cardiac function
Time frame: Between 2 to 3 weeks of life
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Assessment of cardiac function
Time frame: age of 4 and/or 9 months
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Assessment of cardiac function
Time frame: 3, 5 and/or 8 years
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain)by %
Assessment of cardiac function
Time frame: 11, 14 and/or 17 years
Our primary outcome is cardiac function assessed by STE using peak longitudinal strain) by %
6 secondary outcomes
Prevalence of pulmonary hypertension
Time frame: Within 24 hours of life
Prevalence of pulmonary hypertension
Time frame: Between day 3 to 5 of life
Prevalence of pulmonary hypertension
Time frame: Between 2 to 3 weeks of life
Prevalence of pulmonary hypertension
Time frame: age of 4 and/or 9 months
Prevalence of pulmonary hypertension
Time frame: 3, 5 and/or 8 years
Prevalence of pulmonary hypertension
Time frame: 11, 14 and/or 17 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2024.
- Individual participant dataSharing planned: No
Related trials for Congenital Diaphragmatic Hernia
Enrolling by invitationObservational study
North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia
Active, not recruiting
Fetoscopic Endoluminal Tracheal Occlusion in Severe Left Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia, Pulmonary Hypoplasia
Active, not recruitingPhase 2
Effect of a Parenteral Emulsion With Omega3 on Neonates With PPHN and CDH
Pulmonary Hypertension of Newborn, Diaphragm Defect
RecruitingPhase 4
De-implementing Inhaled Nitric Oxide for Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia
Active, not recruiting
Fetoscopic Endoluminal Tracheal Occlusion (FETO) for Severe Left Diaphragmatic Hernia (CDH)
Congenital Diaphragmatic Hernia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Diaphragmatic Hernia, run by McGill University Health Centre/Research Institute of the McGill University Health Centre at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of cardiac function (time frame: Within 24 hours of life). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2024.