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Fetal Endotracheal Occlusion (FETO) in the Resolution of Pulmonary Hypertension in Fetuses With Severe CDH

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ClinicalTrials.gov Identifier: NCT03980717
Recruitment Status : Recruiting
First Posted : June 10, 2019
Last Update Posted : September 11, 2019
Sponsor:
Collaborators:
Baylor College of Medicine
Texas Children's Hospital
Information provided by (Responsible Party):
Michael Belfort, Baylor College of Medicine

Brief Summary:

Congenital diaphragmatic hernia (CDH) occurs when the diaphragm fails to fully fuse and leaves a portal through which abdominal structures can migrate into the thorax. In the more severe cases, the abdominal structures remain in the thoracic cavity and compromise the development of the lungs. Infants born with this defect have a decreased capacity for gas exchange; mortality rates after birth have been reported between 40-60%. Now that CDH can be accurately diagnosed by mid-gestation, a number of strategies have been developed to repair the hernia and promote lung tissue development.

Fetal tracheal occlusion (FETO), using a fetoscopically delivered and removed balloon device, has been used to temporarily occlude the trachea and increase lung distension in CDH to allow the lungs to develop and has been shown to increase survival at birth. The role of FETO in the resolution of pulmonary hypertension in fetuses with severe left- and right- sided CDH remains unclear. Our recent observation that FETO is associated with a higher proportion of infants who resolve their pulmonary hypertension by the age of 1 year as compared with those who have not had FETO, is based on a retrospective cohort study, which, as with any such design, has some intrinsic limitations. Thus, a prospective cohort study that is appropriately powered to confirm or disprove this encouraging observation is needed. If our preliminary observation is confirmed, resolution of PH by the age of 1 year could be added to the benefits of the FETO procedure in severe left and right-sided CDH cases.

The investigators will perform 40 FETO procedures on fetuses diagnosed prenatally with severe right- or left-sided CDH, and outcome data will be compared with that of a control group of severe right- or left-sided CDH who will not undergo the FETO procedure because of medical or social issues. Because the prevalence of left-sided CDH is higher than right-side CDH, the investigators will perform 25 FETO procedures in left sided CDH and 15 in right-sided CDH, and these outcomes will be compared to a cohort of 40 non FETO cases.


Condition or disease Intervention/treatment Phase
Congenital Diaphragmatic Hernia Device: Goldbal Detachable Balloon and delivery microcatheter Not Applicable

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: The investigators will perform 40 FETO procedures on fetuses diagnosed prenatally with severe right- or left-sided CDH and outcome data will be compared with that of a control group of severe right- or left-sided CDH who will not undergo the FETO procedure because of medical or social issues. Because the prevalence of left-sided CDH is higher than right-side CDH, the investigators will perform 25 FETO procedures in left sided CDH and 15 in right-sided CDH, whose outcomes will be compared to a cohort of 40 non FETO cases. The control group will consist of patients who fit the same fetal inclusion/exclusion criteria as our FETO subjects, and will be matched by variables including maternal age, body mass index, gestational age, severity of CDH and site of CDH (left- or right-sided).
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Prospective Study on the Role of Fetal Endotracheal Occlusion (FETO) in the Resolution of Pulmonary Hypertension Among Fetuses With Severe Congenital Diaphragmatic Hernia
Actual Study Start Date : August 9, 2019
Estimated Primary Completion Date : December 2028
Estimated Study Completion Date : December 2030


Arm Intervention/treatment
Experimental: Fetal Endotracheal Occlusion (FETO)
Placement and retrieval of the GoldBAL4 or GoldBAL2 Detachable balloon using the plug/unplug method, using BALTACCIDBPE100 Delivery Catheter.
Device: Goldbal Detachable Balloon and delivery microcatheter
Between 28+0/7 - 31+6/7 weeks gestation for severe CDH placement of the detachable balloon. Balloon retrieval will be planned for no later than 36+6/7 weeks at the discretion of the FETO center.
Other Names:
  • GOLDBAL4 or GOLDBAL2 Goldballoon
  • BALTACCIBDPE100 delivery microcatheter

No Intervention: non-FETO
The control group will consist of patients who did not undergo the FETO procedure who fit the same fetal inclusion/exclusion criteria as our FETO subjects and will be matched by variables including maternal age, body mass index, gestational age, severity of CDH and site of CDH (left- or right-sided).



Primary Outcome Measures :
  1. Resolution of Pulmonary Hypertension - 6 months [ Time Frame: by the age of 6 months ]
    To compare the proportion of fetuses with resolution of PH by the age of 6 months in neonates with severe CDH who underwent the FETO procedure and that of a control group composed of neonates who did not have the FETO procedure.

  2. Resolution of Pulmonary Hypertension - 12 months [ Time Frame: by the age of 12 months ]
    To compare the proportion of fetuses with resolution of PH by the age of 12 months in neonates with severe CDH who underwent the FETO procedure and that of a control group composed of neonates who did not have the FETO procedure.


Secondary Outcome Measures :
  1. 2-year Survival [ Time Frame: 2 years after childbirth ]
    To assess two-year neonatal survival following FETO.

  2. Number of participants who undergo a successful completion of surgical procedures/balloon placement [ Time Frame: Up to 10 weeks ]
    To assess the successful completion of surgical procedures/placement of balloons in fetuses with severe CDH. Case report forms are utilized to record study related data, including any procedural complications such as failed balloon placement or failed balloon retrieval, as well as surgical or anesthesia complications. At least twice a month fetal surveillance will be performed for up to 10 weeks post balloon placement.

  3. Maternal Outcomes- Maternal Morbidity-incidence of preterm delivery [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of preterm delivery (spontaneous or indicated).

  4. Maternal Outcomes- Maternal Morbidity-incidence of cesarean section [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of cesarean section rate.

  5. Maternal Outcomes- Maternal Morbidity-length of hospitalization [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of length of hospitalization after the FETO procedure.

  6. Maternal Outcomes- Maternal Morbidity- length of hospitalization after UNPLUG procedure [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of length of hospitalization after balloon removal (UNPLUG)

  7. Maternal Outcomes- Maternal Morbidity- vaginal bleeding [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of post procedure vaginal bleeding.

  8. Maternal Outcomes- Maternal Morbidity- Placental abruption [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of post-procedure placental abruption.

  9. Maternal Outcomes- Maternal Morbidity- Rupture of membranes [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of post-procedure rupture of membranes. Amniotic fluid level and membrane status will also be monitored at weekly intervals by ultrasonography.

  10. Maternal Outcomes- Maternal Morbidity- oligohydramnios [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of post-procedure oligohydramnios.Amniotic fluid level and membrane status will also be monitored at weekly intervals by ultrasonography.

  11. Maternal Outcomes- Maternal Morbidity- chorioamnionitis [ Time Frame: Up to 6 weeks postpartum ]
    Case report forms are utilized to record study related data through patient's medical chart review. Maternal morbidity will be assessed in terms of incidence of post-procedure chorioamnionitis.

  12. Neonatal/Child outcomes- Pulmonary Morbidity - Number of babies that require ECMO [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Short-term measures of neonatal pulmonary morbidity, including the need for extracorporeal membrane oxygenation.

  13. Neonatal/Child outcomes- Pulmonary Morbidity- ventilatory support [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Short-term measures of neonatal pulmonary morbidity, including the duration of neonatal ventilatory support.

  14. Neonatal/Child outcomes- Pulmonary Morbidity- Number of participants that require the use of supplemental oxygen [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Short-term measures of neonatal pulmonary morbidity, including the need for administration of supplemental oxygen.

  15. Neonatal/Child outcomes- gastrointestinal morbidity [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Short-term measures of gastrointestinal morbidity.

  16. Neonatal/Child outcomes-neurologic morbidity [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Short-term measures of neurologic morbidity.

  17. Neonatal/Child outcomes- Survival to discharge from the hospital [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Survival to discharge from the hospital.

  18. Neonatal/Child outcomes- Duration of hospitalization. [ Time Frame: Up to 2 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Duration of hospitalization after delivery.

  19. Neonatal/Child outcomes- Number of participants that need supplemental oxygen [ Time Frame: Up to 18 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Assessments of measures of long-term morbidity as the need for supplemental oxygen.

  20. Neonatal/Child outcomes- recurrent infection [ Time Frame: Up to 18 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Assessments of measures of long-term morbidity as the rates of recurrent infection.

  21. Neonatal/Child outcomes- repeated hospitalization [ Time Frame: Up to 18 years of age ]
    Case report forms are utilized to record study related data through patient's medical chart. Assessments measures of long-term morbidity as the need for repeated hospitalization. A general health questionnaire will also be given in the form of an interview, either face to face or over the phone. The questionnaire contains questions regarding the children's health status; including hospitalizations, medications, surgical procedures, medical interventions and additional therapies he/she is receiving, and questions to evaluate his/her behavioral and social development.

  22. Neonatal/Child outcomes- Neurodevelopmental [ Time Frame: Up to 18 years of age ]
    Developmental Profile 3 (DP-3) questionnaire will be utilized to record study related data. Assessments of neurodevelopmental outcomes.



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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient is a pregnant woman between 18 and 45 years of age
  • Singleton pregnancy
  • The fetuses will be 28+0/7 to 31+6/7 weeks of gestational age
  • Confirmed diagnosis of severe left- or right-sided CDH of the fetus: Observed/expected total lung volume equal to or less than 0.32 with more than 21% of liver herniated into the hemithorax. (Ideally calculated between 28+0/7 and 31+6/7 weeks' gestation.)
  • Normal fetal echocardiogram or echocardiogram with a minor anomaly (such a small VSD) that in the opinion of the pediatric cardiologist will not affect postnatal outcome
  • Normal fetal karyotype or microarray
  • The mother must be healthy enough to have surgery
  • Patient and father of the baby provide signed informed consent that details the maternal and fetal risks involved with the procedure
  • Patient willing to remain in Houston for the duration following balloon placement until delivery.

Exclusion Criteria:

  • Contraindication to abdominal surgery, fetoscopic surgery, or general anesthesia
  • Allergy to latex
  • Allergy or previous adverse reaction to a study medication specified in this protocol
  • Preterm labor, preeclampsia, or uterine anomaly (e.g., large fibroid tumor) in the index pregnancy.
  • Fetal aneuploidy, known structural genomic variants, other major fetal anomalies that may impact the fetal/neonatal survival or known syndromic mutation
  • Suspicion of major recognized syndrome (e.g. Fryns syndrome) on ultrasound or MRI
  • Maternal BMI >35
  • High risk for fetal hemophilia

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03980717


Contacts
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Contact: Michael Belfort, MD PhD 832-826-7375 belfort@bcm.edu
Contact: Rebecca M Johnson, BA 832-826-7451 rj2@bcm.edu

Locations
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United States, Texas
Texas Childrens Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Michael Belfort, M.D.    832-826-7375      
Contact: Rebecca Johnson, BA    832-826-7451      
Principal Investigator: Michael A Belfort, M.D.         
Sub-Investigator: Alireza A Shamshirsaz, M.D.         
Sub-Investigator: Jimmy Espinoza, M.D.         
Sub-Investigator: Christopher Cassady, M.D.         
Sub-Investigator: Timothy Lee, M.D.         
Sub-Investigator: Magdalena Sanz Cortes, M.D., PhD         
Sub-Investigator: Ahmed Nassr, M.D.         
Sub-Investigator: Wesley Lee, M.D.         
Sub-Investigator: Alice King, M.D.         
Sub-Investigator: Roopali Donepudi, MD         
Sub-Investigator: Sundeep Keswani, MD         
Sponsors and Collaborators
Michael Belfort
Baylor College of Medicine
Texas Children's Hospital
Investigators
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Principal Investigator: Michael Belfort, MD PhD Baylor College of Medicine/Texas Children's Hospital

Publications:

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Responsible Party: Michael Belfort, Chairman and Professor, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT03980717     History of Changes
Other Study ID Numbers: H-42958
First Posted: June 10, 2019    Key Record Dates
Last Update Posted: September 11, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: Yes
Device Product Not Approved or Cleared by U.S. FDA: Yes
Keywords provided by Michael Belfort, Baylor College of Medicine:
Fetal Tracheal Occlusion
FETO plug-unplug
Goldvalve balloon
Severe Congenital Diaphragmatic Hernia
Goldbal balloon
FETO
Additional relevant MeSH terms:
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Hypertension, Pulmonary
Hypertension
Hernias, Diaphragmatic, Congenital
Hernia
Hernia, Diaphragmatic
Vascular Diseases
Cardiovascular Diseases
Pathological Conditions, Anatomical
Lung Diseases
Respiratory Tract Diseases
Congenital Abnormalities