Congenital Diaphragmatic Herniapro-CDHNCT03179371
Proteomic Profiling for Congenital Diaphragmatic Hernia
An observational study of Congenital Diaphragmatic Hernia, run by Centre Hospitalier Universitaire de Besancon at 2 sites in France.
Trial summary
Congenital diaphragmatic hernia (CDH) is a severe congenital malformation, related to a developmental defect of the diaphragm. The incidence of CDH is approximated at 1 in 3,000 live births. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- Congenital Diaphragmatic Hernia
- How many people
- 10 participants
- Where
- 2 sitesFrance
- Study dates
- 18 May 2017to Nov 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 to 50 years
- Women only
- No healthy volunteers
- Adult
Study population: Mothers whose fetus has CDH
Who may be able to join
- Pregnant patients for whom amniocentesis will be performed as part of the prenatal diagnosis of a diaphragmatic hernia.
Who cannot join
- Refusal of patients,
- Refusal of spouse regarding data of the future child,
- Multiple malformations,
- Twin pregnancy,
- Prenatal diagnosis of an unconfirmed diaphragmatic hernia,
- Miscarriage after amniocentesis.
Trial locations and contacts
2 sites in 1 country.
France2 sites, 1 recruiting
- Active, not recruiting
CHU de Besançon
Besançon, 25000
- Recruiting
CHU de Dijon
Dijon, 21000
Thierry ROUSSEAU, MD, PhD, thierry.rousseau@chu-dijon.fr
About this study
Official title: Proteomic Analysis of Amniotic Fluid in the Case of Diaphragmatic Hernia: Search for Prognostic Expression Profiles
Congenital diaphragmatic hernia (CDH) is a severe congenital malformation, related to a developmental defect of the diaphragm. The incidence of CDH is approximated at 1 in 3,000 live births. Although advances in surgery and neonatal intensive care have improved the prognosis, mortality remains high, around 30-50% related to severe lung hypoplasia and persistent pulmonary hypertension. Prenatal evaluation with observed/expected Lung over Head Ratio (o/e LHR), liver position and total lung volume measured by magnetic resonance, have been shown to correlate with neonatal mortality . However, the preponderant factor of persistent pulmonary hypertension remains difficult to predict prenatally. In patients with isolated diaphragmatic hernia (without associated malformations or karyotype abnormalities), prognosis is evaluated indirectly on pulmonary development from pulmonary volume measurements. Apart from the most caricatural cases with extremely good or very pejorative values, for a large proportion of fetuses with diaphragmatic dome hernia the prognosis remains uncertain.
The aim of the proposal is to investigate whether the analysis of the proteom of the amniotic fluid of the fetuses with CDH could give information of a prognostic character. The objective of the study is to identify, from the proteomic profile of the amniotic fluid of mothers whose fetus has CDH, prognostic markers candidates for death at 2 months of the infant. The first step is to carry out an exploratory and non-interventional study on a small sample (n = 10) of the target population. This is a preliminary step before considering, if the results are encouraging, a large-scale study from a biological collection to determine candidate proteins (new biomarkers) which relative expression levels could be used as surrogate marker of pulmonary hypoplasia.
Read the detailed description
Pregnant patients for whom amniocentesis will be performed as part of the prenatal management of CDH confirmed by fetal ultrasound will be approached fot inclusion in the study. The indication of amniocentesis will be indicated according to the usual standards of care. Briefly, the study will not involve any additional invasive procedure. In our study, amniocentesis will be indicated during the conventional management of these patients. The needs of our research will not lead to additional amniocenteses, but simply an increase in the volume of amniotic fluid collected. An additional volume of 5 ml will be sampled for volumes usually taken from 20 to 30 ml. This volume of sampling will have a very limited impact on maternal and fetal well-being. An analysis of the proteom of the amniotic fluid of the patients will be carried out. The relative amounts of proteins and peptides contained in the amniotic fluid will be analyzed to explore variations in proteomic profile that may reflect different clinical gravity in terms of further evolution. The usual management of the patients and their children will not be modified by the procedures related to the research.
The first step will consist in carrying out a depletion of the major proteins, in particular albumin. A protein assay will be carried out from the same amount of protein for each sample (standardization). This depletion step will be followed by a migration step on a gel of 1D electrophoresis. Its purpose is both to eliminate all the contaminants that can hinder mass spectrometry analysis, to separate the proteins according to their molecular weight in order to simplify the protein mixture and to have a visual control of the heterogeneity of the sample. A differential visual analysis after staining will allow to define the highly variable zones between the groups of patients and within the same sample. We will select the zone of interest in identical manner for each sample and for the whole of the gel track. The use of commercial electrophoresis gels of the same batch and the parallel analysis of several samples of different groups and the use of quality controls ensure reproducibility. The proteins contained in these bands / gel zones are then digested according to a standardized protocol under controlled conditions. The simultaneous treatment of the different biological samples makes it possible to considerably reduce the impact of the variability of this step in the final differential statistical analysis. At the end of this step the proteins are analyzed by an LC-ESI MS / MS approach. This LC-ESI MS / MS approach consists of separating the peptides on a chromatographic separation column which is coupled directly to the ESI-MS / M mass spectrometer. This analytical approach can allow the identification of more than 1000 proteins and 5000 peptides. This analysis is repeated 3 times per sample so that analytical variability can be taken into account in differential statistical studies. At the end of this protocol the raw data of each zone are pooled by sample and the differential statistical study can be carried out.
- Conditions
- Congenital Diaphragmatic Hernia
- 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.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Mothers whose fetus has CDH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neonatal death
Measured: 2 months of age
Secondary outcomes (13)
Prenatal death of the fetus
Death during pregnancy
Measured: 9 months
Neonatal death
Measured: 15 days
Apgar score
Measured: 5 min after birth
Age at surgery
Measured: 6 months of age
Need for a diaphragmatic prosthesis
Measured: 6 months of age
Duration of ventilation (in days)
Measured: 6 months of age
Duration of the oxygen dependency (in days)
Measured: 6 months of age
Need of supplemental oxygen therapy
Binary outcome (Yes/No)
Measured: 28 days of age
Occurrence of pulmonary hypertension
Measured: 6 months of age
Duration of parenteral nutrition (in days)
Measured: 6 months of age
Age at the beginning of enteral nutrition
Measured: 6 months of age
Age at the beginning of oral nutrition
Measured: 6 months of age
Duration of hospital stay (in days)
Measured: 6 months of age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2017
- Study start18 May 2017
- Primary completionJul 2019 (estimated)
- Study completionNov 2019 (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.
- 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
- Other
- Time perspective
- Prospective
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Besancon
- Sponsor type
- University, hospital or other
- Collaborators
- FEMTO-ST
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03179371
- Sponsor's protocol ID
- API/2016/78
- Acronym
- pro-CDH
- Status last verified
- Jul 2018
- First submitted
- 16 May 2017
- Last updated
- 18 Jul 2018
- 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 18 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Proteomic Analysis of Amniotic Fluid in the Case of Diaphragmatic Hernia: Search for Prognostic Expression Profiles
- ClinicalTrials.gov ID
- NCT03179371
- Acronym / sponsor ID
- pro-CDH / API/2016/78
- Conditions
- Congenital Diaphragmatic Hernia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de BesanconUniversity, hospital or other
- Collaborators
- FEMTO-ST
- Study start
- 18 May 2017 (actual)
- Primary completion
- Jul 2019 (estimated)
- Study completion
- Nov 2019 (estimated)
- First posted
- 7 Jun 2017 (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.
Congenital diaphragmatic hernia (CDH) is a severe congenital malformation, related to a developmental defect of the diaphragm. The incidence of CDH is approximated at 1 in 3,000 live births. Although advances in surgery and neonatal intensive care have improved the prognosis, mortality remains high, around 30-50% related to severe lung hypoplasia and persistent pulmonary hypertension. Prenatal evaluation with observed/expected Lung over Head Ratio (o/e LHR), liver position and total lung volume measured by magnetic resonance, have been shown to correlate with neonatal mortality . However, the preponderant factor of persistent pulmonary hypertension remains difficult to predict prenatally. In patients with isolated diaphragmatic hernia (without associated malformations or karyotype abnormalities), prognosis is evaluated indirectly on pulmonary development from pulmonary volume measurements. Apart from the most caricatural cases with extremely good or very pejorative values, for a large proportion of fetuses with diaphragmatic dome hernia the prognosis remains uncertain.
The aim of the proposal is to investigate whether the analysis of the proteom of the amniotic fluid of the fetuses with CDH could give information of a prognostic character. The objective of the study is to identify, from the proteomic profile of the amniotic fluid of mothers whose fetus has CDH, prognostic markers candidates for death at 2 months of the infant. The first step is to carry out an exploratory and non-interventional study on a small sample (n = 10) of the target population. This is a preliminary step before considering, if the results are encouraging, a large-scale study from a biological collection to determine candidate proteins (new biomarkers) which relative expression levels could be used as surrogate marker of pulmonary hypoplasia.
Detailed description
Pregnant patients for whom amniocentesis will be performed as part of the prenatal management of CDH confirmed by fetal ultrasound will be approached fot inclusion in the study. The indication of amniocentesis will be indicated according to the usual standards of care. Briefly, the study will not involve any additional invasive procedure. In our study, amniocentesis will be indicated during the conventional management of these patients. The needs of our research will not lead to additional amniocenteses, but simply an increase in the volume of amniotic fluid collected. An additional volume of 5 ml will be sampled for volumes usually taken from 20 to 30 ml. This volume of sampling will have a very limited impact on maternal and fetal well-being. An analysis of the proteom of the amniotic fluid of the patients will be carried out. The relative amounts of proteins and peptides contained in the amniotic fluid will be analyzed to explore variations in proteomic profile that may reflect different clinical gravity in terms of further evolution. The usual management of the patients and their children will not be modified by the procedures related to the research.
The first step will consist in carrying out a depletion of the major proteins, in particular albumin. A protein assay will be carried out from the same amount of protein for each sample (standardization). This depletion step will be followed by a migration step on a gel of 1D electrophoresis. Its purpose is both to eliminate all the contaminants that can hinder mass spectrometry analysis, to separate the proteins according to their molecular weight in order to simplify the protein mixture and to have a visual control of the heterogeneity of the sample. A differential visual analysis after staining will allow to define the highly variable zones between the groups of patients and within the same sample. We will select the zone of interest in identical manner for each sample and for the whole of the gel track. The use of commercial electrophoresis gels of the same batch and the parallel analysis of several samples of different groups and the use of quality controls ensure reproducibility. The proteins contained in these bands / gel zones are then digested according to a standardized protocol under controlled conditions. The simultaneous treatment of the different biological samples makes it possible to considerably reduce the impact of the variability of this step in the final differential statistical analysis. At the end of this step the proteins are analyzed by an LC-ESI MS / MS approach. This LC-ESI MS / MS approach consists of separating the peptides on a chromatographic separation column which is coupled directly to the ESI-MS / M mass spectrometer. This analytical approach can allow the identification of more than 1000 proteins and 5000 peptides. This analysis is repeated 3 times per sample so that analytical variability can be taken into account in differential statistical studies. At the end of this protocol the raw data of each zone are pooled by sample and the differential statistical study can be carried out.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Mothers whose fetus has CDH |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Mothers whose fetus has CDH
Key inclusion criteria (1 listed)
- Pregnant patients for whom amniocentesis will be performed as part of the prenatal diagnosis of a diaphragmatic hernia.
Key exclusion criteria (6 listed)
- Refusal of patients,
- Refusal of spouse regarding data of the future child,
- Multiple malformations,
- Twin pregnancy,
- Prenatal diagnosis of an unconfirmed diaphragmatic hernia,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Neonatal death
Time frame: 2 months of age
13 secondary outcomes
Prenatal death of the fetus
Time frame: 9 months
Neonatal death
Time frame: 15 days
Apgar score
Time frame: 5 min after birth
Age at surgery
Time frame: 6 months of age
Need for a diaphragmatic prosthesis
Time frame: 6 months of age
Duration of ventilation (in days)
Time frame: 6 months of age
Duration of the oxygen dependency (in days)
Time frame: 6 months of age
Need of supplemental oxygen therapy
Time frame: 28 days of age
Occurrence of pulmonary hypertension
Time frame: 6 months of age
Duration of parenteral nutrition (in days)
Time frame: 6 months of age
Age at the beginning of enteral nutrition
Time frame: 6 months of age
Age at the beginning of oral nutrition
Time frame: 6 months of age
Duration of hospital stay (in days)
Time frame: 6 months of age
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2018.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
2 sites 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 Centre Hospitalier Universitaire de Besancon at 2 sites in France.
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 1 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 18 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Frédéric Auber, Professor (+ 33 3 81 21 82 21, fauber@chu-besancon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neonatal death (time frame: 2 months of age). 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 18 Jul 2018.