FETO Therapy for Diaphragmatic Hernia

(FETO Trial)

Overseen By Anthony Johnson, DO

Age: 18 - 65

Sex: Female

Trial Phase: Academic

Sponsor: The University of Texas Health Science Center, Houston

Disqualifiers: Multi-fetal pregnancy, Latex allergy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: From 27 weeks gestational age up to 39 weeks gestational age

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the safety and feasibility of a procedure called Fetoscopic Endoluminal Tracheal Occlusion (FETO) for treating unborn babies with a severe form of left Congenital Diaphragmatic Hernia (CDH). CDH is a birth defect where a hole in the diaphragm allows organs to move into the chest, hindering lung development. The trial will determine if this treatment can be safely used at UTHealth and potentially in larger, multi-center studies. Pregnant women carrying a single baby diagnosed with severe left CDH may be eligible to participate. As an unphased trial, this study offers participants the chance to contribute to groundbreaking research that could lead to new treatment options for CDH.

Do I need to stop my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more information.

Do I have to stop taking my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. It's best to discuss your specific medications with the trial team to get a clear answer.

What prior data suggests that FETO therapy is safe for fetuses with severe left Congenital Diaphragmatic Hernia?

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy is a promising treatment for severe congenital diaphragmatic hernia (CDH) in unborn babies. Studies have found that FETO can lead to good outcomes for these patients.

FETO is generally well-tolerated, but it does carry some risks. Reports have noted tracheomegaly, an enlargement of the windpipe, as a possible side effect. However, many centers have successfully performed the procedure, indicating it is safe enough for further research.

Despite the risks, the potential benefits make FETO a worthwhile option in severe cases of CDH. It is important to discuss any concerns with a healthcare provider. ^{1 2 3 4 5}

Why are researchers excited about this trial?

FETO therapy is unique because it offers a new way to treat congenital diaphragmatic hernia (CDH) by temporarily placing a balloon in the fetus's trachea. Unlike traditional surgical options that occur after birth, FETO focuses on prenatal intervention to promote lung growth before the baby is born. Researchers are excited about this approach because it has the potential to improve lung development early on, potentially increasing survival rates and reducing complications associated with CDH.

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

Research has shown that Fetoscopic Endoluminal Tracheal Occlusion (FETO), the treatment participants in this trial will receive, can aid in severe congenital diaphragmatic hernia (CDH) in unborn babies. Studies indicate that FETO offers positive results for this condition. Data from the TOTAL trials revealed that FETO significantly improves survival rates for babies. Compared to traditional care, FETO is also linked to better brain development by age two. These findings suggest that FETO could be a promising treatment for severe CDH. ^{1 2 4 6 7}

Who Is on the Research Team?

Anthony Johnson

Principal Investigator

The University of Texas Health Science Center, Houston

Are You a Good Fit for This Trial?

This trial is for pregnant women over 18 with a single pregnancy and severe left Congenital Diaphragmatic Hernia (CDH) in the fetus. Eligible participants must have an LHR less than 30%, be able to stay near the hospital, and not have any conditions that risk mother or baby during surgery.

Inclusion Criteria

Singleton pregnancy

My baby's genetic test results are normal.

Patient meets psychosocial criteria

Exclusion Criteria

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

Psychosocial ineligibility, precluding consent: Inability to reside within 30 minutes of The Fetal Center at Children's Memorial Hermann Hospital and inability to comply with the travel for the follow-up requirements of the trial; Patient does not have a support person (e.g. spouse, partner, mother) available to stay with the patient for the duration of the pregnancy at The Fetal Center at Children's Memorial Hermann Hospital

Additional fetal anomaly and chromosomal abnormalities by ultrasound, MRI, or echocardiogram at the fetal treatment center. No cases will be removed post hoc if abnormalities are discovered in the course of post-operative monitoring

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy is performed on fetuses with severe left Congenital Diaphragmatic Hernia (CDH)

From 27 weeks gestational age up to 39 weeks gestational age

Follow-up

Participants are monitored for maternal complications such as preterm labor and chorioamnionitis after the insertion of the balloon until delivery

Up to 12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• FETO therapy

Trial Overview The study tests Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy's safety and feasibility on fetuses with severe CDH. It's a preliminary study at UTHealth to see if they can join larger multi-center trials later.

How Is the Trial Designed?

Group I: FETO therapy Experimental Treatment 1 Intervention

Intervention name: FETO therapy

FETO therapy is already approved in United States for the following indications:

Approved in United States as FETO for:

• Severe congenital diaphragmatic hernia (CDH)

Who Is Running the Clinical Trial?

The University of Texas Health Science Center, Houston

Lead Sponsor

Trials

974

Recruited

361,000+

Anthony Johnson

Lead Sponsor

Trials

1

Recruited

20+

Published Research Related to This Trial

Fetoscopic endoluminal tracheal occlusion (FETO) significantly improved survival rates for infants with severe congenital diaphragmatic hernia, with 40% of infants in the FETO group surviving to discharge compared to only 15% in the expectant care group, based on a trial involving 80 women.

While FETO showed a clear survival benefit, it also increased the risks of complications such as preterm birth and prelabor rupture of membranes, highlighting the need for careful consideration of risks and benefits in prenatal treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized Trial of Fetal Surgery for Severe Left Diaphragmatic Hernia. Deprest, JA., Nicolaides, KH., Benachi, A., et al.[2022]

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]

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]

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]

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]

Fetoscopic endoluminal tracheal occlusion (FETO) was performed on 21 cases of severe congenital diaphragmatic hernia (CDH), resulting in a postnatal survival rate of 47% for infants with isolated unilateral CDH.

The study found that the increase in the observed/expected lung-to-head ratio (O/E LHR) after the FETO procedure was significantly greater in surviving infants compared to those who did not survive, indicating a potential predictor of survival.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fetoscopic Endoluminal Tracheal Occlusion in Fetuses with Severe Diaphragmatic Hernia: A Three-Year Single-Center Experience. Persico, N., Fabietti, I., Ciralli, F., et al.[2018]

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. 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 ...

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

Fetoscopic Endoluminal Tracheal Occlusion-Synergic ... In comparison with severe CDH managed with expectant care, FETO appears to be linked to more favorable neurodevelopmental outcomes at 24 months [57]. A ...

5. nejm.org nejm.org/doi/full/10.1056/NEJMoa2027030

Randomized Trial of Fetal Surgery for Severe Left ... Previous studies have suggested that FETO improves survival among infants with congenital diaphragmatic hernia. Two small, single-center, ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10863657/

Fetoscopic Endoluminal Tracheal Occlusion for Severe, Left ... OBJECTIVE: To report the outcomes of fetoscopic endoluminal tracheal occlusion in a multicenter North American cohort of patients with isolated, ...

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.