FETO Surgery for Congenital Diaphragmatic Hernia

Overseen By Katie Archbold, BA

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: Hanmin Lee

Disqualifiers: Multifetal pregnancy, Preterm labor, Placental abnormalities, Maternal HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Between 27 and 29 weeks gestation

10 Participants Needed

Trial Summary

What is the purpose of this trial?

Despite advances in prenatal diagnosis and postnatal therapies, including ECMO (Extracorporeal Membrane Oxygenation), inhaled nitric oxide therapy, and ventilator strategies that minimize ventilator-induced lung injury, morbidity and mortality rates for babies with severe CDH remain high. The rationale for fetal therapy in severe CDH is to promote adequate lung growth for neonatal survival. Prenatal tracheal occlusion obstructs the normal egress of lung fluid during pulmonary development leading to increased lung tissue stretch, increased cell proliferation, and accelerated lung growth. The investigator's goal with this pilot study is to study the feasibility of implementing Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy in the most severe group of fetuses with left CDH (LHR O/E \< 25%).

Do I need to stop my current medications for the FETO Surgery trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your healthcare provider.

What data supports the effectiveness of the treatment Fetoscopic Endoluminal Tracheal Occlusion (FETO) for Congenital Diaphragmatic Hernia?

Research shows that FETO can improve the survival of infants with severe congenital diaphragmatic hernia (CDH), although there are concerns about potential complications like tracheomegaly (enlarged windpipe) and tracheomalacia (softening of the windpipe). ^{1 2 3 4 5}

How is the FETO treatment different from other treatments for congenital diaphragmatic hernia?

FETO is a unique treatment for congenital diaphragmatic hernia because it involves a minimally invasive procedure where a balloon is placed in the fetus's trachea (windpipe) to promote lung growth before birth, which is different from traditional surgical approaches that are performed after birth. ^{1 2 3 4 5}

Research Team

Hanmin Lee, MD

Principal Investigator

University of California, San Francisco

Eligibility Criteria

This trial is for pregnant women over 18 with a single pregnancy and severe left CDH in the fetus, where the liver has moved into the chest. Participants must have normal fetal chromosomes, be at 27-29 weeks gestation for surgery, meet psychosocial criteria, consent to participate, and secure payment or insurance coverage.

Inclusion Criteria

I am pregnant and 18 years or older.

My unborn baby has a severe lung problem diagnosed by ultrasound.

Gestational age at FETO procedure 27 weeks 0 days to 29 weeks 6 days as determined by clinical information (LMP) and evaluation of first ultrasound

Exclusion Criteria

Placental abnormalities (previa, abruption, accrete) known at time of enrollment

Multi-fetal pregnancy

I do not have HIV or Hepatitis B/C, or I have tested negative for these.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Fetoscopic Endoluminal Tracheal Occlusion (FETO) surgery and removal of balloon using the BALT GOLDBAL2 balloon and BALTACCIBDPE100 catheter

Between 27 and 29 weeks gestation

Follow-up

Participants are monitored for fetal lung growth and survival rate after FETO

6 months after birth

Treatment Details

Interventions

• Fetoscopic Endoluminal Tracheal Occlusion

Trial Overview The study tests Fetoscopic Endoluminal Tracheal Occlusion (FETO) surgery on fetuses with severe lung underdevelopment due to CDH. The goal is to see if blocking the trachea prenatally can improve lung growth before birth.

Participant Groups

Group I: Device - FETO Experimental Treatment 1 Intervention

Fetoscopic Endoluminal Tracheal Occlusion (FETO) surgery and removal of balloon using the BALT GOLDBAL2 balloon and BALTACCIBDPE100 catheter.

Fetoscopic Endoluminal Tracheal Occlusion is already approved in United States, European Union for the following indications:

Approved in United States as FETO for:

• Severe congenital diaphragmatic hernia (CDH)

Approved in European Union as FETO for:

• Severe congenital diaphragmatic hernia (CDH)

Who Is Running the Clinical Trial?

Hanmin Lee

Lead Sponsor

Trials

1

Recruited

10+

Findings from Research

A systematic review of 10 studies involving 449 infants who underwent fetoscopic endoluminal tracheal occlusion (FETO) for congenital diaphragmatic hernia (CDH) found that 6% of infants born alive and 12% of those who survived to discharge experienced symptomatic tracheal complications.

These complications ranged from mild symptoms like barking cough to severe issues requiring tracheostomy, highlighting the need for ongoing monitoring of FETO survivors and the development of devices that reduce tracheal injury.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prevalence of symptomatic tracheal morbidities after fetoscopic endoluminal tracheal occlusion: a systematic review and meta-analysis. Tho, ALW., Rath, CP., Tan, JKG., et al.[2023]

Fetal endoscopic tracheal occlusion (FETO) was performed successfully in 210 cases of congenital diaphragmatic hernia (CDH), with a high success rate of balloon placement (96.7%) and a median procedure duration of 10 minutes.

While FETO is associated with a significant risk of spontaneous preterm prelabor rupture of membranes (47.1%) and preterm delivery, it notably improves survival rates for affected fetuses, increasing survival from 24.1% to 49.1% for left-sided CDH and from 0% to 35.3% for right-sided CDH.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Jani, JC., Nicolaides, KH., Gratacós, E., et al.[2022]

Fetoscopic endoluminal tracheal occlusion (FETO) is a feasible and safe procedure for treating severe congenital diaphragmatic hernia (CDH), with successful balloon insertion in all 11 cases studied, and no severe maternal adverse events reported.

The procedure resulted in a 45% survival rate at 90 days of age and at discharge for the treated fetuses, indicating its potential effectiveness in promoting fetal lung growth in cases of isolated left-sided CDH.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility and outcomes of fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: A Japanese experience. Wada, S., Ozawa, K., Sugibayashi, R., et al.[2020]

In a study of 18 children with severe congenital diaphragmatic hernia (CDH) who underwent fetoscopic endoluminal tracheal occlusion (FETO), long-term survival rates were comparable to a non-FETO group, with 67% surviving at 5 years.

Despite improvements in pulmonary morbidity and gastroesophageal reflux disease, many FETO patients continued to require bronchodilators and feeding tubes, indicating ongoing health challenges even after the procedure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Morbidity in children after fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: Results from a multidisciplinary clinic. Sferra, SR., Nies, MK., Miller, JL., et al.[2023]

In a trial involving 196 women, fetoscopic endoluminal tracheal occlusion (FETO) did not show a significant improvement in survival rates for infants with moderate congenital diaphragmatic hernia compared to expectant care, with 63% survival in the FETO group versus 50% in the control group (P=0.06).

FETO was associated with higher risks of preterm birth and prelabor rupture of membranes (44% vs. 12% and 64% vs. 22%, respectively), indicating potential safety concerns despite no serious maternal complications reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized Trial of Fetal Surgery for Moderate Left Diaphragmatic Hernia. Deprest, JA., Benachi, A., Gratacos, E., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Prevalence of symptomatic tracheal morbidities after fetoscopic endoluminal tracheal occlusion: a systematic review and meta-analysis. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. [2022]

3. Australia pubmed.ncbi.nlm.nih.gov

Feasibility and outcomes of fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: A Japanese experience. [2020]

4. United States pubmed.ncbi.nlm.nih.gov

Morbidity in children after fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: Results from a multidisciplinary clinic. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Randomized Trial of Fetal Surgery for Moderate Left Diaphragmatic Hernia. [2022]