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

25 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.

How is the treatment for congenital diaphragmatic hernia using the GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter different from other treatments?

This treatment, known as Fetoscopic Endoluminal Tracheal Occlusion (FETO), is unique because it involves placing a balloon in the fetus's trachea (windpipe) to help the lungs grow before birth, which is not a standard approach for congenital diaphragmatic hernia. It is a specialized procedure that requires careful timing and skilled removal of the balloon after delivery to prevent airway obstruction. ^{1 2 3 4 5}

What is the purpose of this trial?

CDH is a birth defect characterized by the development, very early in gestation, of a hole in the diaphragm, the breathing muscle that separates the chest from the abdomen. As a result, the intestines and other organs in the abdomen can move into the chest and press on the developing lungs. This prevents the lungs from growing and developing normally. In severe cases, CDH can lead to serious disease and death at birth. For these babies, treatment before birth may allow the lungs to grow enough before birth so these children are capable of surviving and thriving.

Research Team

Holly L Hedrick, MD

Principal Investigator

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

Eligibility Criteria

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.

Psychosocial ineligibility, precluding consent

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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]

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]

References

1. Ireland pubmed.ncbi.nlm.nih.gov

Simulation training for urgent postnatal fetal tracheal balloon removal: Two learning methods. [2023]

2. Ireland pubmed.ncbi.nlm.nih.gov

Tracheal side effects of endoscopic balloon tracheal occlusion in the fetal lamb model. [2019]

3. Australia pubmed.ncbi.nlm.nih.gov

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

4. England pubmed.ncbi.nlm.nih.gov

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

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

Single-Center Outcome of Fetoscopic Tracheal Balloon Occlusion for Severe Congenital Diaphragmatic Hernia. [2021]