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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a procedure called Fetoscopic Endoluminal Tracheal Occlusion (FETO) to determine its safety and effectiveness in treating severe Congenital Diaphragmatic Hernia (CDH), a serious birth defect affecting the diaphragm. Participants will either undergo the FETO procedure or receive the usual care without it. Pregnant individuals with a single baby diagnosed with severe CDH and specific conditions, such as the liver pushing into the chest cavity, may be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.

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 prior data suggests that Fetoscopic Endoluminal Tracheal Occlusion (FETO) is safe for treating severe Congenital Diaphragmatic Hernia?

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO) can be a safe procedure for treating severe Congenital Diaphragmatic Hernia (CDH) in unborn babies. One study found that although FETO did not improve survival rates compared to standard treatments, it was performed safely. Another study indicated that FETO is a viable option for severe CDH cases with reasonable outcomes.

Survival rates after FETO varied based on the type of CDH. For instance, one group had a 64% survival rate, while another had a 33% rate. These findings suggest that while FETO is safe, its success can depend on the specific situation.

Overall, studies have shown that FETO is generally well-tolerated and can be a safe choice for severe CDH, though results can vary. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CDH?

Researchers are excited about Fetoscopic Endoluminal Tracheal Occlusion (FETO) for treating congenital diaphragmatic hernia because it offers a novel approach compared to standard treatments like surgical repair after birth. FETO involves a minimally invasive procedure conducted in the womb, which temporarily blocks the fetal trachea. This occlusion encourages lung growth during pregnancy, potentially improving outcomes for babies born with this condition. Unlike traditional methods that address the issue only after birth, FETO aims to enhance lung development before the baby is born, offering a proactive solution that could lead to better survival rates and reduced complications.

What evidence suggests that Fetoscopic Endoluminal Tracheal Occlusion is effective for severe Congenital Diaphragmatic Hernia?

Research has shown that a procedure called Fetoscopic Endoluminal Tracheal Occlusion (FETO) could be a promising treatment for severe Congenital Diaphragmatic Hernia (CDH). In this trial, participants in the fetal treatment arm will undergo the FETO procedure. Studies have found that babies with severe CDH who undergo the FETO procedure tend to have better survival rates than those who do not. One study showed that the FETO procedure was successfully completed in almost all cases, indicating it is a practical option. The highest level of clinical evidence supports that FETO can improve survival chances for these babies. This suggests FETO could significantly impact the treatment of severe CDH. ^{1 2 4 5 6}

Who Is on the Research Team?

Raphael Sun, MD

Principal Investigator

Oregon Health and Science University

Andrew Chon, MD

Principal Investigator

Oregon Health and Science University

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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 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]

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]

Citations

1. 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.

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

FETO for Congenital Diaphragmatic Hernia In a sub-analysis comparing patients with severe cases, 63 underwent FETO and 43 were managed expectantly. Although survival was similar for ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1521693425000720

Fetal surgery for congenital diaphragmatic hernia in the ... Current level I evidence demonstrates improved survival rates in patients with severe CDH who undergo FETO. •. Survival impact of FETO in moderate CDH before 30 ...

4. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/full/10.1002/pd.6803

Fetal Endoscopic Tracheal Occlusion (FETO) for Left and ... FETO was successfully performed in 47/48 (98%) cases at an average gestational age (GA) of 28.5 (24–31.6) weeks including 22 (47%) moderate LCDH ...

5. 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 ...

6. chop.edu chop.edu/news/childrens-hospital-philadelphia-researchers-find-specialized-procedure-boosts-survival-infants

Children's Hospital of Philadelphia In severe cases, CDH can lead to serious disease and even death. CHOP offers FETO, a fetal surgery procedure, in select cases of the most severe ...