FETO for Congenital Diaphragmatic Hernia

(FETO Trial)

Overseen By Jena L Miller, MD

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: Johns Hopkins University

Disqualifiers: Age < 18, Right-sided hernia, 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new technique called FETO (Fetoscopic Endoluminal Tracheal Occlusion) to assist babies with severe congenital diaphragmatic hernia (CDH). In this condition, a hole in the diaphragm allows organs to move into the chest, hindering lung growth. The trial involves using a tiny balloon to block the baby's windpipe (trachea) before birth, promoting lung development. It specifically targets pregnancies with a baby diagnosed with a severe form of left-sided CDH, where lung development is significantly compromised. Participants will undergo FETO at different pregnancy stages based on the severity of their baby's condition. As an unphased trial, this study provides a unique opportunity to contribute to groundbreaking research that could improve outcomes for future families facing similar challenges.

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 this technique is safe for treating congenital diaphragmatic hernia?

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO) is generally safe and effective for treating severe congenital diaphragmatic hernia (CDH) in unborn babies. Studies have found that FETO can improve survival rates and lead to better outcomes for these babies. However, like any medical procedure, it carries some risks. For example, some reports mention tracheomegaly, where the windpipe becomes unusually large, as a possible side effect. Overall, the treatment appears promising for increasing survival chances in severe CDH cases. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the FETO procedure using the 11540KE and Balt Goldbal 2 balloon because it provides a novel intervention for congenital diaphragmatic hernia (CDH) by using fetoscopic tracheal occlusion. Unlike the standard of care, which typically involves postnatal surgical repair, FETO is performed in utero, potentially improving lung development before birth. This method specifically targets severe and less severe cases of CDH by strategically timing the balloon placement and removal to optimize lung growth during gestation. This innovative approach holds promise for addressing a critical period of lung development, which could lead to better outcomes for affected infants.

What evidence suggests that FETO is effective for congenital diaphragmatic hernia?

Research has shown that fetoscopic endoluminal tracheal occlusion (FETO), which participants in this trial will receive, can significantly improve survival chances for babies with congenital diaphragmatic hernia (CDH). One study found that the survival rate for babies with a specific type of CDH increased from 24% to 49% after FETO treatment. This procedure involves placing a small balloon in the baby's windpipe to promote lung growth before birth. The goal is to reduce pressure on the lungs, allowing them to develop better and function more effectively after birth. Early results indicate that this method can enhance lung growth and increase survival chances for these babies. ^{1 3 6 7 8}

Who Is on the Research Team?

Ahmet Baschat, MD

Principal Investigator

Johns Hopkins University

Are You a Good Fit for This Trial?

This trial is for pregnant women over 18 with a single, anatomically and chromosomally normal fetus diagnosed with severe left-sided congenital diaphragmatic hernia (CDH) and an observed to expected lung to head ratio (O/E LHR) under 30%. It's not for those with right-sided or bilateral CDH, other major anomalies, O/E LHR ≥ 30%, latex allergies, preterm labor risks like a short cervix, or maternal health issues that make surgery risky.

Inclusion Criteria

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

My pregnancy is between 27 and 29 weeks with a specific hormone ratio below 25%.

My fetus is normal in development and chromosomes.

Exclusion Criteria

I am at risk of early labor due to a short cervix or uterine issues.

I have a health condition that prevents me from having surgery during pregnancy.

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

Up to 82 days

Intervention

Participants undergo Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure

Up to 55 days

Weekly visits for ultrasound and monitoring

Delivery and Follow-up

Participants are monitored from delivery until the child reaches 24 months of age

Up to 744 days

Regular follow-up visits including developmental assessments

What Are the Treatments Tested in This Trial?

Interventions

• 11540KE and Balt Goldbal 2 balloon

Trial Overview The trial tests Fetoscopic Endoluminal Tracheal Occlusion (FETO), using the '11540KE' and 'Balt Goldbal 2 balloon', on fetuses with severe CDH. The aim is to improve survival by promoting lung growth before birth. Babies are divided into two groups based on severity: very severe cases (O/E LHR <25%) and less severe cases (O/E LHR between 25% to <30%).

How Is the Trial Designed?

Group I: 11540KE and Balt Goldbal 2 balloon Experimental Treatment 1 Intervention

Patients meeting inclusion criteria will receive fetoscopic tracheal occlusion using the fetoscopy sheath 11540 KE and the Balt Goldbal2 detachable balloon. Participants with an O/E LHR \<25% (severe group) will have FETO completed at 27 weeks + 0 days to 29 weeks + 6 days gestation. Balloon removal is 4-5 weeks after that. Participants with an O/E LHR 25 to \<30% (less severe group) will have FETO completed at 30 weeks + 0 days to 31 weeks + 6 days gestation. Balloon removal is 3 - 4 weeks after that.

11540KE and Balt Goldbal 2 balloon is already approved in European Union, United States for the following indications:

Approved in European Union as FETO for:

• Severe congenital diaphragmatic hernia (CDH)

Approved in United States as FETO for:

• Severe congenital diaphragmatic hernia (CDH)

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials

2,366

Recruited

15,160,000+

KARL STORZ Endoscopy-America, Inc.

Industry Sponsor

Trials

13

Recruited

21,500+

Published Research Related to This Trial

In a study involving 24 medical students, those trained through in-person lectures were significantly faster at setting up instruments for balloon removal in fetal endoluminal tracheal occlusion (FETO) compared to those trained via online video (62 seconds vs 81 seconds).

However, both training methods showed no significant difference in the time taken to locate and intubate the balloon in the trachea, indicating that while in-person training may enhance setup speed, both methods are equally effective for the critical task of balloon removal.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Simulation training for urgent postnatal fetal tracheal balloon removal: Two learning methods. Lehoczky, L., Corroenne, R., Espinoza, J., et al.[2023]

Fetal endoscopic tracheal occlusion (FETO) is a promising intervention for isolated congenital diaphragmatic hernia that can promote lung growth in fetuses at high risk of postnatal death, with demonstrated feasibility and safety in several fetal surgery programs.

While FETO shows an apparent increase in survival rates compared to controls, the most common complication is preterm premature rupture of membranes, which may complicate the procedure and delivery, highlighting the need for further formal trials to evaluate its efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Technical aspects of fetal endoscopic tracheal occlusion for congenital diaphragmatic hernia. Deprest, J., Nicolaides, K., Done', E., et al.[2016]

Fetoscopic tracheal balloon occlusion was successfully performed in 14 women with severe congenital diaphragmatic hernia, leading to significant improvements in the observed/expected lung/head ratio, indicating enhanced lung growth before birth.

The procedure resulted in a high neonatal survival rate of 93% and an 86% survival rate to hospital discharge, demonstrating its feasibility and favorable outcomes for infants despite the need for intensive postnatal care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Single-Center Outcome of Fetoscopic Tracheal Balloon Occlusion for Severe Congenital Diaphragmatic Hernia. Baschat, AA., Rosner, M., Millard, SE., et al.[2021]

In a study of 16 cases of severe congenital diaphragmatic hernia (CDH) treated with fetoscopic endoluminal tracheal occlusion (FETO), the survival rate at 6 months was 50%, indicating a promising outcome for this prenatal intervention.

Survivors of the treatment had earlier balloon insertion and better changes in amniotic fluid levels and lung-to-head ratio compared to those who did not survive, suggesting that earlier intervention may improve outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetal surgery using fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: a single-center experience. Idelson, A., Tenenbaum-Gavish, K., Danon, D., 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]

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 19 fetuses diagnosed with congenital diaphragmatic hernia (CDH), those who underwent fetal endoluminal balloon tracheal occlusion (FETO) had postnatal outcomes comparable to those who were not prenatally studied, suggesting that FETO can be a viable intervention even for cases with poor prognosis.

Among the 12 babies born after FETO, while some experienced significant complications requiring advanced ventilation support, the overall survival and recovery rates were promising, with no postoperative mortality reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of fetal intervention on postnatal management of congenital diaphragmatic hernia. Saura, L., Castañón, M., Prat, J., et al.[2014]

The study evaluated the safety of the endoluminal balloon used in fetoscopic tracheal occlusion (FETO) for congenital diaphragmatic hernias, finding only minor magnetic field interactions and negligible heating (maximum increase of 0.1°C) when exposed to a 3-T MRI.

MRI-related artifacts were limited to a 10 mm radius around the balloon, indicating that surrounding anatomy can still be visualized, suggesting that MRI can be safely performed in patients with this balloon in place.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of Magnetic Resonance Imaging Safety and Imaging Issues Associated with the Occlusion Balloon Used during Fetoscopic Endoluminal Tracheal Occlusion. Victoria, T., Johnson, AM., Adzick, NS., et al.[2019]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7756773/

Feasibility and outcomes of fetoscopic endoluminal ... Data on the prenatal ultrasound findings, MRI findings, FETO and balloon removal procedure findings, adverse events of fetal therapy, delivery, ...

2. karger.com karger.com/fdt/article/doi/10.1159/000548340/934452/The-Outcomes-and-Lung-Changes-of-Fetoscopic

The Outcomes and Lung Changes of Fetoscopic ... Conclusions: FETO can lead to an increased o/e LHR in some but not all CDH fetuses, reflecting fetal lung growth, and be associated with a ...

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

Fetoscopic Endoluminal Tracheal Occlusion-Synergic ... The survival rates in severe cases of left-sided CDH are less than 25% after birth [8,9]. Although diaphragmatic herniation can be surgically corrected, ...

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

Fetal surgery for congenital diaphragmatic hernia in the ... The TOTAL (Tracheal Occlusion to Accelerate Lung Growth) trial demonstrated a significant improvement in survival rates for fetuses with severe CDH following ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06281717?cond=(DIAPHRAGMATIC%20HERNIA)%20OR%20(ZFPM2)&rank=9

Fetal Endotracheal Occlusion (FETO) in Fetuses with ... Congenital diaphragmatic hernia (CDH) affects 1 in 2,200 to 5,000 live births per year. FETO will be performed with the goal of promoting lung growth and ...

6. 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 ...

7. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0022480424006917

Neonatal Airway Management and Outcomes Following ... Fetoscopic endoluminal tracheal occlusion (FETO) has been shown to improve survival and pulmonary hypertension, however, is associated with tracheomegaly.

8. 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.