Fetoscopic Endoluminal Tracheal Occlusion for Congenital Diaphragmatic Hernia

Age: 18+

Sex: Female

Trial Phase: Phase 3

Sponsor: Oregon Health and Science University

Disqualifiers: Multi-fetal pregnancy, Placental abnormalities, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

80 Participants Needed

Trial Summary

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 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, although there are concerns about potential complications like tracheal issues. ^{1 2 3 4 5}

Is fetoscopic endoluminal tracheal occlusion (FETO) generally safe for humans?

Fetoscopic endoluminal tracheal occlusion (FETO) has been used to improve survival in infants with congenital diaphragmatic hernia, but there are concerns about potential complications like tracheomegaly (enlarged windpipe) and tracheomalacia (softening of the windpipe). ^{1 2 3 4 6}

How is the treatment Fetoscopic Endoluminal Tracheal Occlusion (FETO) different from other treatments for congenital diaphragmatic hernia?

Fetoscopic Endoluminal Tracheal Occlusion (FETO) is unique 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 can improve survival rates for severe congenital diaphragmatic hernia. Unlike other treatments that may focus on postnatal care, FETO is performed during pregnancy to address the condition early. ^{1 2 3 4 5}

What is the purpose of this trial?

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

Research Team

Raphael Sun, MD

Principal Investigator

Oregon Health and Science University

Andrew Chon, MD

Principal Investigator

Oregon Health and Science University

Eligibility Criteria

This trial is for pregnant individuals over 18 with a single pregnancy, less than 29 weeks and 6 days along, carrying a baby with severe left Congenital Diaphragmatic Hernia (CDH) and specific lung-to-head ratio measurements or right CDH within certain criteria. Participants must be able to consent, stay near the hospital, have no major health risks or conflicting conditions.

Inclusion Criteria

Able to provide informed consent

Singleton pregnancy

Cervical length by transvaginal ultrasound >= 20 mm within 24 hours of FETO procedure

Exclusion Criteria

I live more than 30 minutes away from OHSU.

I am under 18 years old.

Multi-fetal pregnancy

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

FETO Procedure

Balloon insertion into the fetal trachea between 27 and 30 weeks of gestation, followed by removal at 34 weeks

7 weeks

Neonatal Intensive Care

Evaluate rates of survival to discharge from the NICU

6 months to 1 year

Follow-up

Participants are monitored for long-term mortality and morbidities

24 months

Treatment Details

Interventions

• Fetoscopic Endoluminal Tracheal Occlusion (FETO)

Trial Overview The study tests the safety and effectiveness of Fetoscopic Endoluminal Tracheal Occlusion (FETO), a procedure aimed at treating babies with severe Congenital Diaphragmatic Hernia (CDH) before they are born.

Participant Groups

Group I: Fetal treatment arm Experimental Treatment 1 Intervention

Cases that will undergo Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure.

Group II: Expectant management arm Active Control 1 Intervention

Cases that meet study inclusion criteria but voluntarily decline the Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure. These cases will undergo the standard of clinical care.

Who Is Running the Clinical Trial?

Oregon Health and Science University

Lead Sponsor

Trials

1,024

Recruited

7,420,000+

Findings from Research

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]

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]

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]

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]

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]

References

1. Australia pubmed.ncbi.nlm.nih.gov

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

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

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

3. 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]

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

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

5. England pubmed.ncbi.nlm.nih.gov

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

6. Switzerland pubmed.ncbi.nlm.nih.gov

A novel translational model of percutaneous fetoscopic endoluminal tracheal occlusion - baboons (Papio spp.). [2021]