Safety and Efficacy of FETO in CDH: A Phase III Trial

NCT06884423 | Recruiting DMC FDA Device

• 2 other identifiers: 24-022603G140236
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 2

Brief Summary

Congenital diaphragmatic hernia (CDH) is a birth defect characterized by the development of a hole in the diaphragm, the breathing muscle that separates the chest from the abdomen. As a result, 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.

Conditions

Congenital Diaphragmatic Hernia; Pulmonary Hypoplasia

Keywords

congenital diaphragmatic hernia; tracheal occlusion

Outcome Measures

Primary Outcomes (4)

• Successful placement and removal of the FETO Device

  Evalutation of successful placement and removal of the fetoscopic endoluminal tracheal occlusion (FETO) device in cases of intrathoracic liver herniation with isolated left congenital diaphragmatic hernia (LCDH) with observed/expected lung to head ratio (O/E LHR) \< 30% or isolated right congenital diaphragmatic hernia (RCDH) with O/E LHR \< 45%.

  24 months

• Comparison of neonatal survival rates of LCDH cases between intervention and control groups

  Compare survival to discharge from the neonatal intensive care units (NICU), between fetuses with intrathoracic liver herniation and isolated LCDH with O/E LHR \< 30% that receive FETO procedure performed at 27\^0 to 29\^6 weeks gestation to concurrent patients with intrathoracic liver herniation, isolated LCDH and O/E LRH \< 30% that undergo expectant management.

  24 months

• Comparison of neonatal survival rates of RCDH cases between intervention and control groups

  Compare the neonatal survival rate to discharge from the neonatal intensive care units (NICU), between fetuses with intrathoracic liver herniation, isolated RCDH with O/E LHR \< 45% that undergo FETO procedure performed at 27\^0 to 29\^6 weeks gestation to concurrent patients with intrathoracic liver herniation, isolated RCDH and O/E LHR \< 45% that proceed with expectant management.

  24 months

• Assessment of complications associated with the FETO intervention

  Evaluate the frequency of maternal and fetal complications associated with the FETO intervention.

  24 months

Secondary Outcomes (2)

• Comparison of long-term mortality and morbidity of LCDH cases between intervention and control groups

  24 months

• Comparison of long-term mortality and morbidity of RCDH cases between intervention and control groups

  24 months

Study Arms (2)

FETO in CDH

EXPERIMENTAL

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. Assignment will be non-randomized. 40 maternal/fetal dyads will be enrolled in this arm.

Device: FETO with GoldBAL2 Balloon and BALTACCIBDPE100 Catheter

Expectant Management in CDH

NO INTERVENTION

The control arm includes patients eligible for FETO according to side of defect, O/E LHR, and liver position who undergo expectant management rather than FETO intervention. Assignment will be non-randomized. 40 maternal/fetal dyads will be enrolled in this arm.

Interventions

FETO with GoldBAL2 Balloon and BALTACCIBDPE100 Catheter DEVICE

Fetoscopic Endoluminal Tracheal Occlusion (FETO) in CDH with GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

FETO in CDH

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Pregnant women age 18 years and older
• Singleton pregnancy
• Normal fetal karyotype with confirmation by culture results, whole exome sequencing (WES), whole genome sequencing (WGS), or chromosomal microarray with non-pathologic variants. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is greater than 26 weeks.
• Gestational age at enrollment is prior to 29 weeks 6 days
• Liver is intrathoracic
• Isolated left congenital diaphragmatic hernia (CDH) with observed/expected lung to head ratio (O/E LHR) of less than 30% at enrollment (18\^0 to 29\^5 weeks), or:
• Isolated right CDH with O/E LHR equal to or less than 45% at enrollment (18\^0 to 29\^5 weeks).
• Cervical length by transvaginal ultrasound equal to or greater than 20 mm within 24 hours of fetoscopic endoluminal tracheal occlusion (FETO) procedure
• Patient meets psychosocial criteria
• Informed consent

You may not qualify if:

• Patient is less than 18 years of age
• Multi-fetal pregnancy
• History of natural rubber latex allergy
• Preterm labor, cervix shortened (less than 20 mm at enrollment or within 24 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor, placenta previa
• Psychosocial ineligibility, precluding consent:
• Inability to reside within 30 minutes of the Children's Hospital of Philadelphia (CHOP) 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 CHOP
• Bilateral CDH, isolated left sided CDH with O/E LHR greater than or equal to 30% (measured at 18\^0 to 29\^5 weeks), isolated right sided CDH with O/E LHR greater than 45% (measured at 180 to 295 weeks), as determined by ultrasound
• No Liver herniation into thoracic cavity.
• Additional fetal anomaly by ultrasound, magnetic resonance imaging (MRI), or echocardiogram at the fetal treatment center. Exclude chromosomal abnormalities, associated anomalies recognized to alter survival prognosis (ie. CDH and congenital heart disease) or presence of an underlying genetic syndrome (ie. Fryns).
• Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy
• History of incompetent cervix with or without cerclage
• Placental abnormalities (previa, abruption, accrete) known at time of enrollment.
• Maternal-fetal Rh isoimmunization, Kell sensitization or neonatal alloimmune thrombocytopenia affecting the current pregnancy.
• Maternal HIV, Hepatitis-B, Hepatitis-C status positive because of the increased risk of transmission to the fetus during maternal-fetal surgery. If the patient's HIV or Hepatitis status is unknown, the patient must be tested and found to have negative results before enrollment.

Sponsors & Collaborators

• Holly L Hedrick, MD: lead

Study Sites (2)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

NOT YET RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Related Publications (7)

• Danzer E, Hedrick HL. Controversies in the management of severe congenital diaphragmatic hernia. Semin Fetal Neonatal Med. 2014 Dec;19(6):376-84. doi: 10.1016/j.siny.2014.10.001. Epub 2014 Nov 7.

  PMID: 25454678 BACKGROUND

• Deprest JA, Gratacos E, Nicolaides K, Done E, Van Mieghem T, Gucciardo L, Claus F, Debeer A, Allegaert K, Reiss I, Tibboel D. Changing perspectives on the perinatal management of isolated congenital diaphragmatic hernia in Europe. Clin Perinatol. 2009 Jun;36(2):329-47, ix. doi: 10.1016/j.clp.2009.03.004.

  PMID: 19559323 BACKGROUND

• Jani JC, Nicolaides KH, Gratacos E, Valencia CM, Done E, Martinez JM, Gucciardo L, Cruz R, Deprest JA. Severe diaphragmatic hernia treated by fetal endoscopic tracheal occlusion. Ultrasound Obstet Gynecol. 2009 Sep;34(3):304-10. doi: 10.1002/uog.6450.

  PMID: 19658113 BACKGROUND

• Style CC, Olutoye OO, Belfort MA, Ayres NA, Cruz SM, Lau PE, Shamshirsaz AA, Lee TC, Olutoye OA, Fernandes CJ, Cortes MS, Keswani SG, Espinoza J. Fetal endoscopic tracheal occlusion reduces pulmonary hypertension in severe congenital diaphragmatic hernia. Ultrasound Obstet Gynecol. 2019 Dec;54(6):752-758. doi: 10.1002/uog.20216. Epub 2019 Nov 4.

  PMID: 30640410 BACKGROUND

• Russo FM, Cordier AG, Basurto D, Salazar L, Litwinska E, Gomez O, Debeer A, Nevoux J, Patel S, Lewi L, Pertierra A, Aertsen M, Gratacos E, Nicolaides KH, Benachi A, Deprest J. Fetal endoscopic tracheal occlusion reverses the natural history of right-sided congenital diaphragmatic hernia: European multicenter experience. Ultrasound Obstet Gynecol. 2021 Mar;57(3):378-385. doi: 10.1002/uog.23115.

  PMID: 32924187 BACKGROUND

• Deprest JA, Nicolaides KH, Benachi A, Gratacos E, Ryan G, Persico N, Sago H, Johnson A, Wielgos M, Berg C, Van Calster B, Russo FM; TOTAL Trial for Severe Hypoplasia Investigators. Randomized Trial of Fetal Surgery for Severe Left Diaphragmatic Hernia. N Engl J Med. 2021 Jul 8;385(2):107-118. doi: 10.1056/NEJMoa2027030. Epub 2021 Jun 8.

  PMID: 34106556 BACKGROUND

• Deprest J, Brady P, Nicolaides K, Benachi A, Berg C, Vermeesch J, Gardener G, Gratacos E. Prenatal management of the fetus with isolated congenital diaphragmatic hernia in the era of the TOTAL trial. Semin Fetal Neonatal Med. 2014 Dec;19(6):338-48. doi: 10.1016/j.siny.2014.09.006. Epub 2014 Nov 11.

  PMID: 25447987 BACKGROUND

Related Links

• CHOP Center for Fetal Diagnosis and Treatment
• CHOP Pulmonary Hypoplasia Program

MeSH Terms

Conditions

Hernias, Diaphragmatic, Congenital

Condition Hierarchy (Ancestors)

Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hernia, Diaphragmatic; Internal Hernia; Hernia; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Holly L Hedrick, MD, FACS

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Holly L Hedrick, MD, FACS

CONTACT

1-800-468-8376; hedrick@chop.edu

Annaliese Aarthun

CONTACT

aarthuna@chop.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator, Attending Surgeon, Professor of Surgery

Study Record Dates

• First Submitted: March 13, 2025
• First Posted: March 19, 2025
• Study Start: April 7, 2025
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): April 1, 2032
• Last Updated: July 25, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

US (2)