Fetal Surgery for Congenital Diaphragmatic Hernia

(FETO Trial)

Recruiting at 1 trial location

Overseen By Katie E Boyle, MPH

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: Connecticut Children's Medical Center

Disqualifiers: Maternal medical condition, Preterm labor, 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: 2 weeks

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Fetoscopic Endoluminal Tracheal Occlusion (FETO) for babies developing with a severe condition known as congenital diaphragmatic hernia (CDH), which affects lung growth before birth. The goal is to help the lungs grow enough for the baby to survive after birth. The trial seeks pregnant women carrying a single baby diagnosed with a severe form of left-sided CDH, indicated by specific ultrasound findings. Participants should also be able to stay at the trial site during key phases of the treatment and care. As an unphased trial, it offers a unique opportunity to contribute to groundbreaking research that could improve outcomes for babies with CDH.

Will I have to stop taking my current medications?

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 doctor.

What prior data suggests that Fetoscopic Endoluminal Tracheal Occlusion (FETO) is safe for fetal surgery?

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO) can be a safe treatment for severe congenital diaphragmatic hernia (CDH) in unborn babies. This procedure promotes better lung growth before birth, increasing the chances of survival afterward.

Studies have found that FETO is minimally invasive, involving less cutting and generally posing fewer risks than traditional surgeries. Previous studies suggest that most patients tolerate the procedure well. However, like any medical procedure, risks exist. Some studies have reported issues related to the procedure, but the potential benefits for babies with severe CDH often outweigh these risks.

Anyone considering this treatment should discuss all potential risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike standard treatments for congenital diaphragmatic hernia (CDH), which often involve postnatal surgery and intensive care, Fetoscopic Endoluminal Tracheal Occlusion (FETO) is a prenatal intervention. FETO is unique because it involves placing a tiny balloon in the fetus's trachea to promote lung growth before birth. This innovative approach directly targets the underdeveloped lungs, potentially improving outcomes by enhancing lung function earlier than traditional methods. Researchers are excited about FETO as it represents a proactive strategy, aiming to optimize lung development and reduce complications associated with CDH.

What evidence suggests that Fetoscopic Endoluminal Tracheal Occlusion (FETO) might be an effective treatment for congenital diaphragmatic hernia?

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO) effectively treats severe congenital diaphragmatic hernia (CDH). Studies have found that FETO promotes lung growth and improves survival chances for babies with this condition. This minimally invasive surgery occurs before birth, where a small balloon is temporarily placed in the baby's windpipe to aid lung development. Previous patients have demonstrated good results with this treatment. Two small studies also suggested that FETO increases survival rates in babies with CDH. Participants in this trial will undergo the FETO procedure to further evaluate its effectiveness. ^{1 2 3 6 7}

Who Is on the Research Team?

Timothy Crombleholme, MD

Principal Investigator

Connecticut Children's Medical Center

Are You a Good Fit for This Trial?

This trial is for pregnant women aged 18 or older with a single pregnancy and a fetus diagnosed with severe left-sided Congenital Diaphragmatic Hernia (CDH) where the liver has moved up into the chest. The baby must have significant lung underdevelopment, confirmed by ultrasound before 29 weeks + 6 days of gestation, and a normal karyotype.

Inclusion Criteria

I am a pregnant woman, 18 or older, and can give consent.

My unborn baby has been diagnosed with a left-sided CDH and the liver is positioned upwards.

Gestation at enrollment prior to 29 wks plus 6 days

Exclusion Criteria

I am pregnant and under 18.

My diaphragmatic hernia is right-sided, bilateral, or a severe left-sided with specific lung-to-head ratio.

I cannot have surgery using a scope due to technical reasons.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure is performed to promote lung growth in fetuses with severe left congenital diaphragmatic hernia

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including postnatal mechanical ventilator support

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Fetoscopic Endoluminal Tracheal Occlusion (FETO)

Trial Overview The trial is testing Fetoscopic Endoluminal Tracheal Occlusion (FETO), which is a prenatal surgery aiming to improve lung growth in fetuses with severe CDH by temporarily blocking their trachea.

How Is the Trial Designed?

Group I: Fetoscopic Endoluminal Tracheal Occlusion (FETO) Experimental Treatment 1 Intervention

An un-blinded non-randomized single arm pilot study of FETO in fetuses with congenital diaphragmatic hernia (CDH)

Fetoscopic Endoluminal Tracheal Occlusion (FETO) 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?

Connecticut Children's Medical Center

Lead Sponsor

Trials

76

Recruited

30,000+

Medical City Children's Hospital

Lead Sponsor

Trials

3

Recruited

60+

Published Research Related to This Trial

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]

The study successfully established a percutaneous fetoscopic endoluminal reversible tracheal occlusion (FETO) model in non-human primates, which is crucial for improving fetal outcomes in cases of congenital diaphragmatic herniation.

The FETO procedure did not result in any complications such as bleeding or premature fetal membrane rupture, and it led to a significant increase in tracheal width in the treated group compared to controls, indicating its potential efficacy in mimicking human conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A novel translational model of percutaneous fetoscopic endoluminal tracheal occlusion - baboons (Papio spp.). Mari, G., Deprest, J., Schenone, M., et al.[2021]

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]

In a study of 129 patients assessed for severe isolated congenital diaphragmatic hernia (iCDH), significant discrepancies were found between initial evaluations and assessments at the fetal surgery center, with 33% showing discordant lung size and 10% having undiagnosed associated anomalies.

Despite the high eligibility for fetoscopic endoluminal tracheal occlusion (FETO) surgery (94%), 26% of parents chose to terminate the pregnancy, highlighting the complexity of decision-making in cases of iCDH.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinically relevant discordances identified after tertiary reassessment of fetuses with isolated congenital diaphragmatic hernia. Done, E., Gucciardo, L., Van Mieghem, T., et al.[2022]

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]

Fetal endoscopic tracheal occlusion (FETO) is a treatment for severe congenital diaphragmatic hernia that can lead to complications such as tracheomegaly in infants, as observed in a series of five cases.

This study highlights a new potential risk associated with FETO, suggesting that the compliant fetal airway may be damaged by balloon occlusion, warranting careful evaluation of the trachea in infants who underwent this procedure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tracheomegaly: a complication of fetal endoscopic tracheal occlusion in the treatment of congenital diaphragmatic hernia. McHugh, K., Afaq, A., Broderick, N., et al.[2021]

Citations

1. mdpi.com mdpi.com/1422-0067/26/4/1639

Fetoscopic Endoluminal Tracheal Occlusion-Synergic ... ... Outcomes in Children After Fetoscopic Endoluminal Tracheal Occlusion for Severe Congenital Diaphragmatic Hernia: Results From a Multidisciplinary Clinic. J ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11627046/

Feasibility, safety, and outcome of fetoscopic endoluminal ... Our data have shown that FETO represents a viable option for severe type of CDH fetuses with reasonable outcome.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06946576

Safety and Efficacy of Fetoscopic Endoluminal Tracheal ... The purpose of this study is to evaluate the safety and efficacy of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in cases of severe Congenital Diaphragmatic ...

4. childrenscolorado.org childrenscolorado.org/advances-answers/recent-articles/fetoscopic-endoluminal-tracheal-occlusion/

FETO for Congenital Diaphragmatic Hernia FETO is a minimally invasive fetal surgery aimed to enhance lung growth and improve survival for patients with severe congenital diaphragmatic hernia (CDH).

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7613453/

Randomized Trial of Fetal Surgery for Severe Left ... Previous studies have suggested that FETO improves survival among infants with congenital diaphragmatic hernia. Two small, single-center, randomized trials ...

6. ajog.org ajog.org/article/S0002-9378(22)00944-9/fulltext

Fetoscopic endoluminal tracheal occlusion (FETO) versus ... Infant survival at 6 months was the primary outcome compared between FETO and expectantly managed cohorts. Secondary outcomes included FETO procedure-related ...

7. cincinnatichildrens.org cincinnatichildrens.org/service/f/fetal-care/conditions/congenital-diaphragmatic-hernia/feto

Fetoscopic Endoluminal Tracheal Occlusion (FETO) for CDH FETO is a surgical procedure to treat the most severe cases of congenital diaphragmatic hernia (CDH) that are diagnosed in utero.