Fetal Endoscopic Tracheal Occlusion (FETO) for Severe Congenital Diaphragmatic Hernia (FETO)

A Study of the Effectiveness of Fetal Endoscopic Tracheal Occlusion (FETO) in the Management of Severe Prenatally Diagnosed Congenital Diaphragmatic Hernia (CDH)

The purpose of the study is to study the efficacy of fetal endoscopic tracheal occlusion (FETO) in cases of severe congenital diaphragmatic hernia (CDH). This study will also collect safety and effectiveness data for the off-label use of the FETO Goldballoon (the balloon that will be inserted into the fetal trachea), manufactured by Balt medical. The investigators hope to study the risks and benefits of FETO in cases of severe CDH in an advanced medical center such as Lucile Packard Children's Hospital (LPCH) Stanford with access to advanced maternal-fetal medicine, neonatal services, and neonatal ECMO, and pediatric surgery.

Study Overview

• Status: Recruiting
• Conditions: Congenital Diaphragmatic Hernia
• Intervention / Treatment: Device: Balt "Goldballoon"

• Study Type: Interventional
• Enrollment (Anticipated): 15
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305-6070
      • Recruiting
      • Lucile Packard Children's Hospital
      • Contact: Christine Lee; Phone Number: 650-725-5720; Email: mfmresearch@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Maternal Age: 18-50
• Singleton gestation
• Gestational age before 29 weeks 6 days
• Severe left or right-sided CDH: For severe left sided CDH observed-to-expected lung-to- head ratio (o/e LHR) less than 25% between 22 and 29 6/7 weeks' gestation, liver herniation, MRI lung volumes less than 30% expected based on gestational age nomograms. For severe right sided CDH o/e LHR less than 30 % between 22 and 29 6/7 weeks' gestation, liver herniation, MRI lung volumes less than 30% expected based on gestational age nomograms.
• Normal genetic karyotype or microarray testing by amniocentesis or chorionic villus sampling (CVS)
• Absence of associated fetal structural cardiac anomalies by a dedicated fetal echocardiogram
• Absence of other structural anomalies by ultrasound or MRI
• Appropriate multi-disciplinary counseling performed with maternal-fetal medicine, neonatology, pediatric surgery, genetics, pediatric otolaryngology (ENT).
• Must be willing to remain near LPCH Stanford (within 30 minutes from the hospital) for the duration of the balloon placement.
• No maternal and/or fetal contra-indications to fetal surgery such as a bleeding disorder, poorly controlled diabetes or hypertension, short cervix (measuring < 20mm), risk for preterm birth etc.
• Planned pregnancy surveillance at LPCH Stanford
• Planned delivery at LPCH Stanford
• Able to provide written consent
• Willingness to comply with all study procedures and availability (meets psychosocial criteria) for the duration of the study including having a support person

Exclusion Criteria:

• Contraindications to fetal surgery including poorly controlled hypertension, diabetes or other maternal medical condition including hematological disorder
• High risk for preterm labor and/or delivery based on either significant history of preterm birth, short cervix (measuring < 20mm), significant uterine anomaly or other risk factor, incompetent cervix (requiring cerclage)
• Non-isolated CDH - CDH with additional structural anomalies
• Significant maternal obesity defined as a body mass index (BMI) greater than 40. BMI is a calculation which includes a person's height and weight
• History of natural rubber latex allergy
• Uterine anomaly such as large or multiple fibroids or mullerian duct abnormality
• Participation in another intervention study that influences maternal and fetal morbidity and mortality.
• Bilateral CDH, left-sided CDH with O/E LHR >25%, or left-sided CDH with O/E LHR <25% but liver completely down in abdomen
• Right-sided CDH O/E LHR >30% or right-sided CDH with O/E LHR <30% with liver completely down in abdomen
• Significant placental abnormalities (abruption, chorioangioma, accreta) known at the time of enrollment and/or surgery
• Maternal-fetal Rhesus isoimmunization, Kell sensitization or neonatal alloimmune thrombocytopenia, affecting the current pregnancy.
• Maternal HIV, Hepatitis B with positive surface antigen, Hepatitis C with presence of virus in maternal blood due to risk of fetal transmission during the procedure
• No safe or feasible fetoscopic approach to balloon placement.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Fetal Endoscopic Tracheal Occlusion (FETO); FETO is performed in-utero and the balloon is removed prior to delivery, and children will have follow-up visits until the age of 2	Device: Balt "Goldballoon"; Fetal endoscopic tracheal occlusion (FETO) is a procedure where a balloon is placed in the main airway or "windpipe" of the fetus, to help with the development of the lungs. This is performed using a fetoscope, a small camera that is inserted into the amniotic cavity and deploying the FETO Goldballoon into the fetal trachea.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of neonates who survive until initial hospital discharge	Average approximately 6 months (up to 1 year)

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of neonates who survive until 28 days of life	28 days
Number of children who survive until 6 months of age	6 months
Number of children who survive until 1 year of age	1 year
Number of children who survive until 2 years of age	2 years
Number of children who receive extracorporeal membrane oxygenation (ECMO)	Average approximately 6 months (up to 1 year)

Collaborators and Investigators

• Sponsor: Yair Blumenfeld
• Investigators: Principal Investigator: Yair Blumenfeld, MD, Stanford University

Study record dates

Study Major Dates

• Study Start (Actual): April 17, 2023
• Primary Completion (Anticipated): August 31, 2025
• Study Completion (Anticipated): August 31, 2027

Study Registration Dates

• First Submitted: July 13, 2022
• First Submitted That Met QC Criteria: July 13, 2022
• First Posted (Actual): July 18, 2022

Study Record Updates

• Last Update Posted (Actual): April 25, 2023
• Last Update Submitted That Met QC Criteria: April 21, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Congenital Abnormalities; Pathological Conditions, Anatomical; Internal Hernia; Hernia; Hernias, Diaphragmatic, Congenital; Hernia, Diaphragmatic
• Other Study ID Numbers: 63230

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes