FETO for Congenital Diaphragmatic Hernia

(FETO Trial)

Overseen By Caitlin Clifford, MD

Age: 18+

Sex: Female

Trial Phase: Academic

Sponsor: Dr Erin Perrone

Disqualifiers: Multi-fetal pregnancy, Latex allergy, Preterm labor, 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: 2

10 Participants Needed

Trial Summary

What is the purpose of this trial?

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 Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100) at Michigan Medicine. The study will enroll pregnant women that meet study criteria. Participants will have placement of FETO between gestational age at 27 weeks plus 0 days and 29 weeks 6 days. The timing for removal of FETO will ideally be between 34 weeks 0 days and 34 weeks and 6 days but ultimately decided by the Fetal Diagnosis and Treatment Center at Michigan Medicine. This study requires that study participants live within 30 miles of the Von Voigtlander Women's Hospital and C.S. Mott Children's Hospital in order to maintain weekly follow up appointments while the balloon is in place and up to delivery. Additionally, there are lifestyle considerations where participants would be unable to carry on normal daily activities including exercise and sexual intercourse, not be able to work the remainder of the pregnancy, as well as have a support person that is available to stay with such as a spouse, friend, partner, parent.

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 data supports the effectiveness of the treatment FETO for Congenital Diaphragmatic Hernia?

Research suggests that FETO (fetal endoscopic tracheal occlusion) can improve survival rates and lung development in babies with severe congenital diaphragmatic hernia, although it may lead to complications like preterm delivery. The treatment is considered promising, but further formal trials are needed to fully evaluate its effectiveness. ^{1 2 3 4 5}

How is the FETO treatment for congenital diaphragmatic hernia unique?

FETO (Fetal Endoscopic Tracheal Occlusion) is unique because it involves placing a balloon in the fetus's trachea (windpipe) to promote lung growth before birth, which is not a standard approach for treating congenital diaphragmatic hernia. This method aims to improve lung development and increase survival rates, unlike traditional treatments that focus on managing symptoms after birth. ^{2 3 6 7 8}

Research Team

Erin Perrone, MD

Principal Investigator

University of Michigan

Eligibility Criteria

This trial is for pregnant women with a baby diagnosed with severe Congenital Diaphragmatic Hernia (CDH). Participants must live within 30 miles of the Von Voigtlander Women's Hospital, have support throughout pregnancy, and be willing to follow study procedures without working or engaging in normal activities like exercise.

Inclusion Criteria

Willing to comply with restrictions of daily living including inability to exercise, have intercourse, or return to work.

Willing to reside within 30 minutes of Von Voigtlander Women's Hospital and ability to maintain follow up appointments

Meets psychosocial criteria

Exclusion Criteria

My baby has a diaphragmatic hernia, with specific lung and liver positioning.

Maternal contraindications to elective fetoscopic surgery

Maternal isoimmunization or neonatal alloimmune thrombocytopenia

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

FETO Procedure

Placement of the Goldballoon Detachable Balloon (GOLDBAL2) in the airway of the fetus between 27 weeks 0 days and 29 weeks 6 days of gestation

2-3 weeks

1 visit (in-person)

Balloon Monitoring

Weekly follow-up appointments to monitor the balloon and fetal lung growth while the balloon is in place

5-7 weeks

Weekly visits (in-person)

Balloon Removal

Removal of the balloon ideally between 34 weeks 0 days and 34 weeks 6 days of gestation

1 week

1 visit (in-person)

Follow-up

Participants are monitored for maternal complications and infant survival up to 180 days after delivery

up to 180 days postpartum

Treatment Details

Interventions

• FETO with Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100)

Trial Overview The trial tests Fetal Endoscopic Tracheal Occlusion (FETO) using a Goldballoon Detachable Balloon and Delivery Microcatheter on babies with CDH. It aims to see if this treatment is safe and doable at Michigan Medicine between 27-29 weeks' gestation.

Participant Groups

Group I: FETO with GOLDBAL2 Experimental Treatment 1 Intervention

A balloon will be placed in the airway of the fetus during the FETO procedure.

FETO with Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100) is already approved in United States, European Union for the following indications:

Approved in United States as FETO for:

• Severe Congenital Diaphragmatic Hernia (CDH)

Approved in European Union as FETO for:

• Severe Congenital Diaphragmatic Hernia (CDH)

Who Is Running the Clinical Trial?

Dr Erin Perrone

Lead Sponsor

Trials

1

Recruited

10+

Findings from Research

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]

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) 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]

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]

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]

Fetoscopic endoluminal tracheal occlusion (FETO) significantly improved lung growth and respiratory function in lambs with congenital diaphragmatic hernia (CDH), as evidenced by increased lung-to-body-weight ratio and dynamic lung compliance.

Despite these improvements, FETO did not lead to significant enhancements in gas exchange during the first two hours of life, indicating that while it aids lung development, further benefits in oxygenation may be limited.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of tracheal occlusion on the neonatal cardiopulmonary transition in an ovine model of diaphragmatic hernia. DeKoninck, PLJ., Crossley, KJ., Kashyap, AJ., 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]

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. Switzerland 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. [2016]

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

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

3. Germany pubmed.ncbi.nlm.nih.gov

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

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

Technical aspects of fetal endoscopic tracheal occlusion for congenital diaphragmatic hernia. [2016]

6. England pubmed.ncbi.nlm.nih.gov

Effects of tracheal occlusion on the neonatal cardiopulmonary transition in an ovine model of diaphragmatic hernia. [2019]

7. Australia pubmed.ncbi.nlm.nih.gov

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

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

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