Tracheal Occlusion for Congenital Diaphragmatic Hernia

(CHOP_FETO Trial)

Overseen By Jennifer T Davis, MSN, RN, CBC

Age: 18 - 65

Sex: Female

Trial Phase: Academic

Sponsor: Children's Hospital of Philadelphia

Disqualifiers: Preterm labor, Placenta previa, 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: 4

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a treatment for babies with Congenital Diaphragmatic Hernia (CDH), a severe condition where a hole in the diaphragm allows organs to move into the chest, hindering lung development. Researchers are testing a procedure called Fetoscopic Endoluminal Tracheal Occlusion (FETO), which places a tiny balloon in the unborn baby's airway to promote lung growth before birth. This procedure uses the GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter. The trial seeks pregnant women with a single fetus diagnosed with severe left-sided CDH, whose babies have very underdeveloped lungs. As an unphased trial, this study aims to explore innovative treatments that could significantly improve outcomes for affected babies.

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 the FETO device is safe for treating CDH?

Research has shown that the Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure is a promising treatment for severe congenital diaphragmatic hernia (CDH) in unborn babies. This procedure uses a special balloon and catheter to promote lung growth by allowing fluid to build up in the airway. Studies have found that FETO is generally well-tolerated. Although all medical procedures carry some risks, evidence supports the safety of this treatment in aiding babies with underdeveloped lungs due to CDH. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the Fetoscopic Endoluminal Tracheal Occlusion (FETO) for congenital diaphragmatic hernia because of its innovative approach. Unlike traditional treatments that rely on postnatal surgical repair, FETO involves placing a detachable balloon in the fetal airway to promote lung growth before birth. This method aims to improve lung development in utero, potentially leading to better breathing outcomes after birth. The use of the GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter provides a targeted, minimally invasive option that could significantly change the outlook for affected babies.

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

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO), a procedure participants in this trial will undergo, can aid babies with congenital diaphragmatic hernia (CDH). In FETO, doctors place a small balloon in the unborn baby's airway, causing fluid to build up in the lungs, which can promote growth. Studies have found that this technique improves lung development in babies with severe CDH. Early results suggest that FETO increases the chances of survival and leads to better health at birth. Although still under study, this method shows promise for treating CDH. ^{1 2 3 4 5}

Who Is on the Research Team?

Holly L Hedrick, MD

Principal Investigator

Children's Hospital of Philadelphia and the Center for Fetal Diagnosis and Treatment

Are You a Good Fit for This Trial?

This trial is for pregnant women over 18 with a single pregnancy and a fetus diagnosed with severe left Congenital Diaphragmatic Hernia (CDH), where the liver has moved up. The baby must have an O/E LHR less than 25%, indicating serious lung underdevelopment, and be no more than 29 weeks + 5 days into gestation.

Inclusion Criteria

My unborn baby's genetic tests are normal.

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

Singleton pregnancy

Exclusion Criteria

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

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

I cannot stay at the hospital for the duration of my treatment and aftercare.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Prenatal Evaluation

Standard prenatal evaluation including medical history, physical exam, ultrasound, echocardiogram, MRI, and psychosocial assessment

1-2 weeks

Multiple visits (in-person)

FETO Procedure

Balloon placement in the fetal airway between 27+0/7 - 29+6/7 gestational age, with removal between 34+0/7 - 34+6/7 gestational age

7 weeks

Weekly monitoring visits (in-person)

Postnatal Stabilization and Surgery

Postnatal stabilization and subsequent surgery to repair the diaphragm at CHOP

Immediate post-birth period

Follow-up

Infants are monitored at 6 months, 12 months, and 2 years of age, and then long-term in the Pulmonary Hypoplasia Program

2 years

3 visits (in-person) and long-term follow-up

What Are the Treatments Tested in This Trial?

Interventions

• GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

Trial Overview The study tests a procedure using GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter to temporarily block the trachea of fetuses in utero. This may help lungs grow by keeping fluid within them, potentially improving survival rates after birth for babies with severe CDH.

How Is the Trial Designed?

Group I: FETO in CDH Experimental Treatment 1 Intervention

Fetoscopic Endoluminal Tracheal Occlusion (FETO) will be performed by placing a detachable balloon inside the fetal airway and removing the balloon after several weeks. Devices: GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter is already approved in United States, European Union for the following indications:

Approved in United States as GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter for:

• Severe left congenital diaphragmatic hernia (CDH)

Approved in European Union as GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter for:

• Severe left congenital diaphragmatic hernia (CDH)

Who Is Running the Clinical Trial?

Children's Hospital of Philadelphia

Lead Sponsor

Trials

749

Recruited

11,400,000+

Published Research Related to This Trial

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]

In a fetal lamb model, in-utero tracheal occlusion using a balloon led to significant dilation of the trachea and mild epithelial changes, including loss of the typical folding pattern and local inflammatory responses, indicating that the procedure effectively obstructs airflow as intended.

When the balloon was removed after two weeks, the tracheal epithelium showed much less damage and inflammation, suggesting that early intervention can allow for recovery and minimize long-term effects on the airway structure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tracheal side effects of endoscopic balloon tracheal occlusion in the fetal lamb model. Deprest, JA., Evrard, VA., Verbeken, EK., et al.[2019]

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 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]

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. clinicaltrials.gov clinicaltrials.gov/study/NCT05771688?term=AREA%5BConditionSearch%5D(%22Hernia,%20Diaphragmatic%22)&rank=3

Fetoscopic Endoluminal Tracheal Occlusion (FETO) The goal of the FETO device is to block the airway with a balloon-type device, allowing fluid to build up in the lungs and help the unborn baby's lungs grow.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT02875860?term=NCT02875860&rank=1

'TOTAL' (Tracheal Occlusion To Accelerate Lung Growth) ... In this study FETO (where GoldBal2 detachable balloon and Baltaccidbpe100 Delivery Catheter are used) is to be done between 30 weeks plus 0 day and 31 weeks ...

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

Feasibility, safety, and outcome of fetoscopic endoluminal ... Feasibility, safety, and outcome of fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia at a low case-load ...

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

The Fall Out of the 2017 European Medical Device Regulation ... The use of the Balt Goldbal-balloon and Baltacci-catheter in Fetoscopic Endoluminal Tracheal Occlusion is affected by the 2017-European Medical ...

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT04052828/fetal-endoscopic-tracheal-occlusion-feto-trial-for-congenital-diaphragmatic-hernia-cdh

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 ...