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

22 Participants Needed

Trial Summary

What is the purpose of this trial?

The goal is to assess the feasibility and safety of implementing Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy in fetuses with severe left Congenital Diaphragmatic Hernia (CDH) at UTHealth. UTHealth's success in this study (with an initial 5 patients) will determine the feasibility of UTHealth's future participation in multi-center trials of this intervention.

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 data supports the idea that FETO Therapy for Diaphragmatic Hernia is an effective treatment?

The available research shows that FETO therapy can improve the survival rates of infants with severe congenital diaphragmatic hernia (CDH). Studies have noted that FETO is promising for severe cases, as it may help the lungs grow better, which is crucial for survival. However, there are some concerns about potential complications, such as issues with the trachea and preterm delivery. Despite these concerns, the treatment is associated with increased survival, especially in severe cases of CDH on the left side. ^{1 2 3 4 5}

What data supports the effectiveness of the treatment FETO therapy for diaphragmatic hernia?

Research shows that FETO therapy can improve the survival of infants with severe congenital diaphragmatic hernia by promoting better lung growth and development, although it may lead to complications like tracheomegaly (enlarged windpipe) and preterm delivery. ^{1 2 3 4 5}

What safety data is available for FETO therapy in treating diaphragmatic hernia?

FETO therapy, used for treating severe congenital diaphragmatic hernia, has been studied for its feasibility and outcomes. Safety concerns include potential complications such as tracheomegaly, tracheomalacia, and preterm delivery. A systematic review and meta-analysis have highlighted these tracheal morbidities. Research also includes developing translational models to improve fetal outcomes and assess new clinical approaches. ^{1 2 3 4 6}

Is FETO therapy generally safe for humans?

FETO therapy, used for treating severe congenital diaphragmatic hernia, has shown some safety concerns, including risks of tracheomegaly (enlarged trachea), tracheomalacia (softening of the trachea), and complications related to preterm delivery. ^{1 2 3 4 6}

Is FETO therapy a promising treatment for diaphragmatic hernia?

Yes, FETO therapy is a promising treatment for diaphragmatic hernia because it has been shown to improve the survival of infants with this condition by helping their lungs grow and develop better before birth. ^{1 2 3 7 8}

How is FETO therapy different from other treatments for diaphragmatic hernia?

FETO therapy is unique because it involves placing a balloon in the unborn baby's trachea (windpipe) to help the lungs grow better before birth, which is different from other treatments that usually occur after birth. This prenatal approach aims to improve lung development and increase survival rates for babies with severe congenital diaphragmatic hernia. ^{1 2 3 7 8}

Research Team

Anthony Johnson

Principal Investigator

The University of Texas Health Science Center, Houston

Eligibility Criteria

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

My unborn baby has a diaphragm issue on the right side, both sides, or isolated left with specific lung measurement.

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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]

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]

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]

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]

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]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Tracheomegaly: a complication of fetal endoscopic tracheal occlusion in the treatment of congenital diaphragmatic hernia. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Prevalence of symptomatic tracheal morbidities after fetoscopic endoluminal tracheal occlusion: a systematic review and meta-analysis. [2023]

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

Randomized Trial of Fetal Surgery for Moderate Left Diaphragmatic Hernia. [2022]

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

Randomized Trial of Fetal Surgery for Severe Left Diaphragmatic Hernia. [2022]

6. Switzerland pubmed.ncbi.nlm.nih.gov

A novel translational model of percutaneous fetoscopic endoluminal tracheal occlusion - baboons (Papio spp.). [2021]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Fetoscopic Endoluminal Tracheal Occlusion in Fetuses with Severe Diaphragmatic Hernia: A Three-Year Single-Center Experience. [2018]

8. Germany pubmed.ncbi.nlm.nih.gov

Fetal surgery using fetoscopic endoluminal tracheal occlusion for severe congenital diaphragmatic hernia: a single-center experience. [2023]