Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia

(FETO Trial)

Age: 18 - 65

Sex: Female

Trial Phase: Academic

Sponsor: Yair Blumenfeld

Disqualifiers: Hypertension, Diabetes, Obesity, 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: Duration not specified

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called fetal endoscopic tracheal occlusion (FETO) for babies diagnosed with severe congenital diaphragmatic hernia (CDH) before birth. The procedure places a small balloon in the baby's windpipe to aid lung development, which is then removed before delivery. The trial uses the Balt Goldballoon to assess the treatment's safety and effectiveness. Pregnant individuals whose baby has severe CDH and who can stay near Stanford for the treatment might be suitable candidates for this trial. As an unphased study, this trial offers a unique opportunity to contribute to groundbreaking research that could improve outcomes for babies with severe 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 fetal endoscopic tracheal occlusion is safe for severe congenital diaphragmatic hernia?

Research has shown that fetal endoscopic tracheal occlusion (FETO) is a promising treatment for severe congenital diaphragmatic hernia (CDH). Studies have found that FETO can reduce the risk of death and improve lung function in affected babies.

In terms of safety, early trials have tested FETO to assess its tolerance. These studies generally found it practical and safe. Although some negative side effects have been reported, they are considered manageable in the medical settings where the procedure occurs.

FETO involves using a special balloon called the Goldballoon, which is placed in the baby's windpipe and removed before birth. Safety data collected so far suggests that with proper care and monitoring, the procedure is well-tolerated.

Participants and their families should discuss the potential risks and benefits with their healthcare providers to make an informed decision about joining a trial. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about Fetal Endoscopic Tracheal Occlusion (FETO) because it offers a unique in-utero intervention for congenital diaphragmatic hernia (CDH) that isn't available with current treatments. Unlike the standard postnatal surgeries, FETO involves placing a balloon in the fetus's trachea before birth, which helps the lungs develop better by blocking the airway and encouraging lung growth. This technique is promising because it targets lung development directly in the womb, potentially improving outcomes before the baby is even born.

What evidence suggests that fetal endoscopic tracheal occlusion is effective for severe congenital diaphragmatic hernia?

Research has shown that fetal endoscopic tracheal occlusion (FETO), which participants in this trial will undergo, can significantly improve survival chances for babies with congenital diaphragmatic hernia (CDH). Studies from the TOTAL trials indicate that FETO enhances lung growth before birth, leading to better health outcomes. The procedure involves placing a small balloon in the unborn baby's windpipe to aid lung development. Specifically, previous patients with severe CDH who underwent FETO had a better chance of survival. This suggests that FETO could be a promising treatment for severe CDH cases. ^{1 2 6 7 8}

Who Is on the Research Team?

Yair Blumenfeld

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for pregnant women aged 18-50 with a single fetus diagnosed with severe congenital diaphragmatic hernia (CDH). They must stay near LPCH Stanford, plan to deliver there, and have normal genetic testing results. Excluded are those with latex allergies, certain CDH measurements, placental abnormalities, maternal infections like HIV or Hepatitis B/C, high risk of preterm labor/delivery, significant obesity (BMI >40), or involvement in another study affecting outcomes.

Inclusion Criteria

Absence of associated fetal structural cardiac anomalies by a dedicated fetal echocardiogram

Absence of other structural anomalies by ultrasound or MRI

Able to provide written consent

Exclusion Criteria

My condition involves a diaphragmatic hernia along with other structural anomalies.

High risk for preterm labor and/or delivery

I do not have uncontrolled high blood pressure, diabetes, or other serious health issues.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Fetal Endoscopic Tracheal Occlusion (FETO) is performed in-utero and the balloon is removed prior to delivery

Duration not specified

Follow-up

Children will have follow-up visits until the age of 2 to monitor safety and effectiveness

2 years

What Are the Treatments Tested in This Trial?

Interventions

• Balt Goldballoon

Trial Overview The FETO Goldballoon procedure by Balt medical is being tested for its effectiveness in treating severe CDH in fetuses. The balloon will be inserted endoscopically into the fetal trachea at LPCH Stanford which has specialized maternal-fetal medicine and neonatal services including ECMO and pediatric surgery.

How Is the Trial Designed?

Group I: Fetal Endoscopic Tracheal Occlusion (FETO) Experimental Treatment 1 Intervention

FETO is performed in-utero and the balloon is removed prior to delivery, and children will have follow-up visits until the age of 2

Balt Goldballoon is already approved in European Union, United States for the following indications:

Approved in European Union as FETO Goldballoon for:

• Severe Congenital Diaphragmatic Hernia (CDH)

Approved in United States as FETO Goldballoon for:

• Investigational use for Severe Congenital Diaphragmatic Hernia (CDH)

Who Is Running the Clinical Trial?

Yair Blumenfeld

Lead Sponsor

Trials

1

Recruited

20+

Published Research Related to This Trial

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]

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]

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) 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]

Endoscopic tracheal occlusion (TO) in fetal lambs with congenital diaphragmatic hernia (CDH) successfully increased lung growth, with 5 out of 11 procedures resulting in live births and expanded lungs.

The TO group showed significantly greater lung weight compared to those without TO, suggesting that this minimally invasive technique could be a promising alternative to open fetal surgery for improving outcomes in CDH.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tracheal obstruction in experimental diaphragmatic hernia: an endoscopic approach in the fetal lamb. Benachi, A., Dommergues, M., Delezoide, AL., et al.[2014]

In a randomized trial involving 24 pregnant women with fetuses diagnosed with severe congenital diaphragmatic hernia, fetal endoscopic tracheal occlusion did not improve survival rates compared to standard postnatal care, with survival rates of 73% and 77% respectively.

The intervention group experienced significantly more complications, including premature rupture of membranes and earlier delivery, indicating that tracheal occlusion may pose additional risks without providing survival benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized trial of fetal endoscopic tracheal occlusion for severe fetal congenital diaphragmatic hernia. Harrison, MR., Keller, RL., Hawgood, SB., et al.[2022]

Fetal endoscopic tracheal occlusion (FETO) using a 1.0-mm fetoscope was successfully performed in 16 fetuses with severe congenital diaphragmatic hernia (CDH), leading to a significant increase in neonatal survival rates (52.9% in the FETO group vs. 5.6% in the control group).

The study also found that FETO reduced the incidence of severe pulmonary arterial hypertension in infants, with 47.1% of the FETO group affected compared to 88.9% in the control group, indicating a potential improvement in neonatal outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparison between fetal endoscopic tracheal occlusion using a 1.0-mm fetoscope and prenatal expectant management in severe congenital diaphragmatic hernia. Ruano, R., Duarte, SA., Pimenta, EJ., et al.[2016]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8192998/

Fetal endoscopic tracheal occlusion for congenital ... The objective of this review is to provide the reader with information on the history of fetal intervention for CDH, current fetal endoscopic tracheal occlusion ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05354505

Fetal Endoscopic Tracheal Occlusion (FETO) Trial for ... This is a single site, single arm, non-randomized, pilot trial to assess the feasibility and safety of treating severe congenital diaphragmatic hernia (CDH) ...

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

Fetal Endoscopic Tracheal Occlusion (FETO) for Left and ... Data from the TOTAL (tracheal occlusion to accelerate lung growth) trials have demonstrated significant improvement in survival following fetal ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04052828?term=AREA%5BConditionSearch%5D(FETO%20CDH)&rank=7

Fetal Endoscopic Tracheal Occlusion (FETO) Trial for ... This is a single site pilot trial to assess the feasibility and safety of treating severe CDH with Fetal Endoscopic Tracheal Occlusion with the Goldballoon ...

5. 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.

6. 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, ...

7. obgyn.onlinelibrary.wiley.com obgyn.onlinelibrary.wiley.com/doi/10.1002/pd.6763

The Fall Out of the 2017 European Medical Device Regulation ... Additionally, a systematic review and meta-analysis confirmed that FETO decreases mortality and pulmonary hypertension in severe CDH and reduces ...

8. stanfordhealthcare.org stanfordhealthcare.org/trials/f/NCT05461222.html

Fetal Endoscopic Tracheal Occlusion (FETO) for Severe ... This study will also collect safety and effectiveness data for the off-label use of the FETO Goldballoon (the balloon that will be inserted into the fetal ...