Congenital Diaphragmatic HerniaPhase 2NCT01731509
Early FETO for Severe Congenital Diaphragmatic Hernia
A Phase 2 study testing Fetal endoscopic tracheal occlusion in people with Congenital Diaphragmatic Hernia, run by University of Sao Paulo General Hospital at 1 site in Brazil.
Trial summary
Congenital diaphragmatic hernia (CDH) is associated high mortality and morbidity, mainly in those cases with severe forms where there are extremely reduced lung volumes, liver herniation and decreased abnormal pulmonary vascularization. Read more about this study
- Who is it for?
- Ages 22 to 28 weeks
- Conditions the study is for
- Congenital Diaphragmatic Hernia
- What is being tested?
- Fetal endoscopic tracheal occlusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteBrazil
- Study dates
- Jun 2014to Dec 2016
- Phase
- Phase 2
- 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 22 to 28 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Fetuses with isolated congenital diaphragmatic hernia (normal fetal karyotype and absence of any associated structural anomaly);
- Gestational age established by last menstruation and/or first trimester ultrasonography;
- Prenatal diagnosis of congenital diaphragmatic hernia before 24 weeks of gestation
- Severe congenital diaphragmatic hernia (at 24 weeks, lung-to-head ratio <1.0 and at least 1/3 of the liver herniated into the fetal thorax)
- written informed consent (by the patient)
Who cannot join
- Preterm premature rupture of the membranes before randomization
- Preterm labor before randomization
Trial locations and contacts
1 site in 1 country.
Brazil1 site
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, São Paulo, 05403-010
About this study
Official title: "Early" Versus "Standard" Fetal Endoscopic Tracheal Occlusion for Severe Congenital Diaphragmatic Hernia - a Randomized Controlled Trial
Congenital diaphragmatic hernia (CDH) is associated high mortality and morbidity, mainly in those cases with severe forms where there are extremely reduced lung volumes, liver herniation and decreased abnormal pulmonary vascularization. Fetal endoscopic tracheal occlusion performed between 26 and 30 weeks (standard FETO) has been shown to increase fetal pulmonary size and vascularity, and to improve infant survival in isolated severe CDH. Fetal pulmonary response followed FETO can be used to predict outcome and is dependent on the size of the fetal lung prior to the procedure.
We hypothesize that performing an earlier FETO, between 22-24 weeks, fetuses with severe form of CDH will have a better fetal pulmonary response and higher chance of surviving.
Read the detailed description
We pretend to investigate if "early FETO" will improve the survival rate and the fetal pulmonary response, by conducting a randomized controlled trial comparing the results with those fetuses that undergo to "standard FETO" (between 26-28 weeks).
- Conditions
- Congenital Diaphragmatic Hernia
- Being tested
- Fetal endoscopic tracheal occlusion
- Keywords
- congenital diaphragmatic hernia, fetoscopy, fetal endoscopic tracheal occlusion, pulmonary hypoplasia, fetal surgery, fetal lung
- 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.
Fetal endoscopic tracheal occlusionOther
Also called FETO or fetal endoscopic traqueal occlusion
FETO will be performed by placing a detachable balloon inside fetal trachea
| Study group | Role | What they receive |
|---|---|---|
| Standard FETO | Active comparator | Group of fetus that undergo fetal endoscopic tracheal occlusion between 26 0/7 weeks and 28 6/7 weeks. Receives: Other: Fetal endoscopic tracheal occlusion |
| Early FETO | Experimental | Group of fetus that undergo fetal endoscopic tracheal occlusion between 22 0/7 weeks and 24 6/7 weeks. Receives: Other: Fetal endoscopic tracheal occlusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Infant survival rate
Percentage of survivors at 6 months of life
Measured: 6 months of life
Secondary outcomes (2)
Postnatal severe pulmonary arterial hypertension (PAH)
Severe PAH will be considered when the neonate presents with profound cyanosis associated with echocardiographic continuous right-to-left shunting through a persistent 'ductus arteriosus' and a persistent difference in pre- to postductal saturation gradient >20%, despite the use of intake Nitric Oxide (iNO).
Measured: 30 days of life
Respiratory morbidity
Need for ventilatory support and/or oxygen dependency.
Measured: 6 months of life
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2012 (estimated)
- Study startJun 2014
- Primary completionDec 2016 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Congenital Diaphragmatic HerniaStudies of the same condition, open and under way.
- More trials run by University of Sao Paulo General HospitalThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- University of Sao Paulo
- Responsible party
- Sponsor
- Investigators
- Rodrigo Ruano, MD PhD, Principal investigator, Faculdade de Medicina da Universidade de Sao Paulo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01731509
- Sponsor's protocol ID
- 8353/12
- Status last verified
- Nov 2012
- First submitted
- 14 Nov 2012
- Last updated
- 17 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Ruano R, Duarte SA, Pimenta EJ, Takashi E, da Silva MM, Tannuri U, Zugaib M. Comparison between fetal endoscopic tracheal occlusion using a 1.0-mm fetoscope and prenatal expectant management in severe congenital diaphragmatic hernia. Fetal Diagn Ther. 2011;29(1):64-70. doi: 10.1159/000311944. Epub 2010 Apr 10.
PubMed 20389048Ruano R, Yoshisaki CT, da Silva MM, Ceccon ME, Grasi MS, Tannuri U, Zugaib M. A randomized controlled trial of fetal endoscopic tracheal occlusion versus postnatal management of severe isolated congenital diaphragmatic hernia. Ultrasound Obstet Gynecol. 2012 Jan;39(1):20-7. doi: 10.1002/uog.10142. Epub 2011 Dec 14.
PubMed 22170862Ruano R, da Silva MM, Campos JA, Papanna R, Moise K Jr, Tannuri U, Zugaib M. Fetal pulmonary response after fetoscopic tracheal occlusion for severe isolated congenital diaphragmatic hernia. Obstet Gynecol. 2012 Jan;119(1):93-101. doi: 10.1097/AOG.0b013e31823d3aea.
PubMed 22183216Harrison MR, Keller RL, Hawgood SB, Kitterman JA, Sandberg PL, Farmer DL, Lee H, Filly RA, Farrell JA, Albanese CT. A randomized trial of fetal endoscopic tracheal occlusion for severe fetal congenital diaphragmatic hernia. N Engl J Med. 2003 Nov 13;349(20):1916-24. doi: 10.1056/NEJMoa035005.
PubMed 14614166Jani JC, Nicolaides KH, Gratacos E, Valencia CM, Done E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.
PubMed 19658113Deprest J, Gratacos E, Nicolaides KH; FETO Task Group. Fetoscopic tracheal occlusion (FETO) for severe congenital diaphragmatic hernia: evolution of a technique and preliminary results. Ultrasound Obstet Gynecol. 2004 Aug;24(2):121-6. doi: 10.1002/uog.1711.
PubMed 15287047
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- "Early" Versus "Standard" Fetal Endoscopic Tracheal Occlusion for Severe Congenital Diaphragmatic Hernia - a Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT01731509
- Acronym / sponsor ID
- 8353/12
- Conditions
- Congenital Diaphragmatic Hernia
- Keywords
- congenital diaphragmatic hernia, fetoscopy, fetal endoscopic tracheal occlusion, pulmonary hypoplasia, fetal surgery, fetal lung
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- University of Sao Paulo General HospitalUniversity, hospital or other
- Collaborators
- University of Sao Paulo
- Study start
- Jun 2014
- Primary completion
- Dec 2016 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 21 Nov 2012 (estimated)
- 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 associated high mortality and morbidity, mainly in those cases with severe forms where there are extremely reduced lung volumes, liver herniation and decreased abnormal pulmonary vascularization. Fetal endoscopic tracheal occlusion performed between 26 and 30 weeks (standard FETO) has been shown to increase fetal pulmonary size and vascularity, and to improve infant survival in isolated severe CDH. Fetal pulmonary response followed FETO can be used to predict outcome and is dependent on the size of the fetal lung prior to the procedure.
We hypothesize that performing an earlier FETO, between 22-24 weeks, fetuses with severe form of CDH will have a better fetal pulmonary response and higher chance of surviving.
Detailed description
We pretend to investigate if "early FETO" will improve the survival rate and the fetal pulmonary response, by conducting a randomized controlled trial comparing the results with those fetuses that undergo to "standard FETO" (between 26-28 weeks).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard FETO | Active comparator | Other: Fetal endoscopic tracheal occlusionGroup of fetus that undergo fetal endoscopic tracheal occlusion between 26 0/7 weeks and 28 6/7 weeks. |
| Early FETO | Experimental | Other: Fetal endoscopic tracheal occlusionGroup of fetus that undergo fetal endoscopic tracheal occlusion between 22 0/7 weeks and 24 6/7 weeks. |
Who may qualify
- Ages
- 22 to 28 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Fetuses with isolated congenital diaphragmatic hernia (normal fetal karyotype and absence of any associated structural anomaly);
- Gestational age established by last menstruation and/or first trimester ultrasonography;
- Prenatal diagnosis of congenital diaphragmatic hernia before 24 weeks of gestation
- Severe congenital diaphragmatic hernia (at 24 weeks, lung-to-head ratio <1.0 and at least 1/3 of the liver herniated into the fetal thorax)
- written informed consent (by the patient)
Key exclusion criteria (2 listed)
- Preterm premature rupture of the membranes before randomization
- Preterm labor before randomization
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 |
|---|---|---|
| Fetal endoscopic tracheal occlusionAlso: FETO or fetal endoscopic traqueal occlusionFETO will be performed by placing a detachable balloon inside fetal trachea | Other | Early FETO, Standard FETO |
Primary outcome
Infant survival rate
Time frame: 6 months of life
Percentage of survivors at 6 months of life
2 secondary outcomes
Postnatal severe pulmonary arterial hypertension (PAH)
Time frame: 30 days of life
Respiratory morbidity
Time frame: 6 months of life
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2013.
- Background: PubMed 20389048Ruano R, Duarte SA, Pimenta EJ, Takashi E, da Silva MM, Tannuri U, Zugaib M. Comparison between fetal endoscopic tracheal occlusion using a 1.0-mm fetoscope and prenatal expectant management in severe congenital diaphragmatic hernia. Fetal Diagn Ther. 2011;29(1):64-70. doi: 10.1159/000311944. Epub 2010 Apr 10.
- Background: PubMed 22170862Ruano R, Yoshisaki CT, da Silva MM, Ceccon ME, Grasi MS, Tannuri U, Zugaib M. A randomized controlled trial of fetal endoscopic tracheal occlusion versus postnatal management of severe isolated congenital diaphragmatic hernia. Ultrasound Obstet Gynecol. 2012 Jan;39(1):20-7. doi: 10.1002/uog.10142. Epub 2011 Dec 14.
- Background: PubMed 22183216Ruano R, da Silva MM, Campos JA, Papanna R, Moise K Jr, Tannuri U, Zugaib M. Fetal pulmonary response after fetoscopic tracheal occlusion for severe isolated congenital diaphragmatic hernia. Obstet Gynecol. 2012 Jan;119(1):93-101. doi: 10.1097/AOG.0b013e31823d3aea.
- Background: PubMed 14614166Harrison MR, Keller RL, Hawgood SB, Kitterman JA, Sandberg PL, Farmer DL, Lee H, Filly RA, Farrell JA, Albanese CT. A randomized trial of fetal endoscopic tracheal occlusion for severe fetal congenital diaphragmatic hernia. N Engl J Med. 2003 Nov 13;349(20):1916-24. doi: 10.1056/NEJMoa035005.
- Background: PubMed 19658113Jani JC, Nicolaides KH, Gratacos E, Valencia CM, Done E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.
- Background: PubMed 15287047Deprest J, Gratacos E, Nicolaides KH; FETO Task Group. Fetoscopic tracheal occlusion (FETO) for severe congenital diaphragmatic hernia: evolution of a technique and preliminary results. Ultrasound Obstet Gynecol. 2004 Aug;24(2):121-6. doi: 10.1002/uog.1711.
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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.
Rodrigo Ruano, MD PhD principal investigator
Faculdade de Medicina da Universidade de Sao Paulo
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fetal endoscopic tracheal occlusion in people with Congenital Diaphragmatic Hernia, run by University of Sao Paulo General Hospital at 1 site in Brazil.
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 5 inclusion and 2 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 17 Dec 2013. 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 Brazil. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Rodrigo Ruano, MD PhD ((5511)95739188, rodrigoruano@usp.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Infant survival rate (time frame: 6 months of life). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01731509, which also lists 6 linked publications.
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 17 Dec 2013.