Infant Survival and Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia, A Phase III Trial

NCT06179472 | Active Not Recruiting DMC FDA Device

• 1 other identifier: STUDY00003414
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to determine the infant survival and long-term effects of performing Fetoscopic Endoluminal Tracheal Occlusion (FETO) surgery and removal of the BALT Goldbal2 balloon at Children's Mercy Hospital. We hypothesize that FETO balloon placement may increase survival and decrease morbidity when compared to standard prenatal care for the treatment of severe left or right congenital diaphragmatic hernia (CDH).

Conditions

Congenital Diaphragmatic Hernia

Outcome Measures

Primary Outcomes (3)

• Infant survival

  Infant survival will be measured from delivery to discharge, at 6, 12, and 24 months of age.

  Birth to 24 months

• Number of occurrences and severity of pulmonary hypertension

  Recording the occurrence of severe pulmonary hypertension based on echocardiogram

  Birth to 6 months

• Use of Extracorporeal Membrane Oxygenation (ECMO) support

  Number of infants requiring ECMO support

  Birth to 6 months

Secondary Outcomes (5)

• Change in fetal lung growth

  The first ultrasound within 1 week after balloon removal (balloon removal ideally at 34 weeks gestation)

• Maternal Complications

  Up to 4-6 weeks post-partum

• Gestational age at delivery

  At the time of delivery

• Number of infants requiring supplemental oxygen

  Birth to 24 months

• Number of days in neonatal intensive care unit (NICU) / pediatric intensive care unit (PICU)

  Birth to 1 year

Study Arms (2)

Balloon Arm

EXPERIMENTAL

Participants who consent to the insertion and the removal of the Balt GOLDBAL2 Balloon.

Device: BALT GOLDBAL2 Balloon

No Treatment

NO INTERVENTION

Patients who have been diagnosed with severe CDH at our site but choose not to participate in the study.

Interventions

BALT GOLDBAL2 Balloon DEVICE

Placement and retrieval of the GOLDBALLOON Detachable Balloon for severe CDH

Balloon Arm

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability (meets psychosocial criteria below) for the duration of the study
• Pregnant persons age 18 years and older
• Singleton pregnancy
• Absence of life-limiting genetic anomalies on microarray or karyotype on amniocentesis. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is \> 26 weeks gestation
• Fetal echocardiogram with changes expected with CDH and no major structural cardiac defects (small ventricular septal defect (VSD)/atrial septal defect (ASD) may be included as they will not alter outcome and are not life-limiting)
• Left or Right CDH with liver up without presence of concomitant life-limiting anomalies.
• Gestational age for Left-CDH at enrollment prior to 29 weeks plus 6 days for O/E LHR \<30%; for Right-CDH with O/E LHR \<45% gestational age at enrollment prior to 29 weeks plus 6 days.
• Meets psychosocial criteria
• Willing to reside within 30 minutes of Children's Mercy Hospital Fetal Health Center and ability to maintain follow up appointments
• Patient has a support person (e.g. spouse, partner, friend, or parent) that is available to stay with her for the duration of the pregnancy.
• Willing to comply with restrictions of daily living including inability to exercise, have intercourse or return to work.

You may not qualify if:

• Pregnant persons \< 18 years of age
• Multi-fetal pregnancy
• Rubber latex allergy
• History of preterm labor, cervix shortened (\<15mm at enrollment or within 24 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor, placenta previa
• Psychosocial ineligibility precluding consent:
• Inability to reside within 30 minutes of Children's Mercy Hospital Fetal Health Center until balloon removal or delivery (if clinically indicated)
• Patient does not have a support person (e.g., spouse, partner, parent) available to stay with the patient until balloon removal or duration of the pregnancy (if clinically indicated)
• Adult unable to consent
• Prisoners
• Social work or health psychology will meet with each patient to evaluate the social situation and support system. Identifiable issues of social instability or compliance with the protocol will exclude the patient as a potential candidate.
• Bilateral CDH, isolated left-sided with O/E LHR ≥30% (measured at 18 weeks 0 days to 29 weeks 5 days) as determined by ultrasound, isolated right-sided CDH with O/E LHR ≥45% (measured at 18 years 0 days to 29 weeks 5 days) as determined by ultrasound.
• Additional life-limiting fetal anomaly by ultrasound, MRI or echocardiogram that may affect outcome. These include chromosomal or anatomic abnormalities, associated anomalies recognized to alter survival prognosis (i.e., CDH and severe congenital heart disease) or presence of an underlying genetic syndrome (e.g., Fryns). No cases will be removed post hoc if abnormalities are discovered in the course of post-operative monitoring.
• Patient contraindication to fetoscopic surgery or severe medical condition in pregnancy
• History of incompetent cervix with or without cerclage
• Placental abnormalities (previa, abruption, accreta) known at the time of enrollment

Sponsors & Collaborators

• NAFTNet FETO Consortium: collaborator
• University of Wisconsin, Madison: collaborator
• Inna Lobeck: lead

Study Sites (1)

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

MeSH Terms

Conditions

Hernias, Diaphragmatic, Congenital

Condition Hierarchy (Ancestors)

Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hernia, Diaphragmatic; Internal Hernia; Hernia; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Inna Lobeck

  Physician

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Physician

Model Details: Comparison of outcomes between patients who elect to receive the intervention versus those that do not in the specified time-frame

Study Record Dates

• First Submitted: November 27, 2023
• First Posted: December 22, 2023
• Study Start: April 14, 2024
• Primary Completion (Estimated): January 1, 2031
• Study Completion (Estimated): January 1, 2032
• Last Updated: March 19, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)