Fetoscopic Endoluminal Tracheal Occlusion in Severe Left Congenital Diaphragmatic Hernia (CHOP_FETO)

Pilot Trial of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in Severe Left Congenital Diaphragmatic Hernia (CDH)

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.

Study Overview

• Status: Recruiting
• Conditions: Congenital Diaphragmatic Hernia; Pulmonary Hypoplasia
• Intervention / Treatment: Device: GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

Detailed Description

Study Summary

All patients will complete a standard prenatal evaluation at the Center for Fetal Diagnosis and Treatment (CFDT) to find out if they are candidates. The standard clinical assessments include: Medical history and Physical exam, Level II ultrasound, Fetal echocardiogram, Fetal magnetic resonance imaging (MRI), and a Psychosocial assessment.

If determined eligible, patients will be extensively counseled by the CFDT Team and those who choose to participate will provide written, informed consent for study enrollment.

Up to 25 women will be enrolled in the FETO study. The mother and her unborn baby will undergo two procedures. A balloon will be placed in the unborn baby's airway between 27+0/7 - 29+6/7 gestational age. The balloon blocks the airway and remains in place until balloon removal. The timing for balloon removal will be determined by the CFDT Management Team and can occur between 34 +0/7 - 34+6/7 gestational age.

Mothers enrolled in this study must remain near the fetal center, under close supervision, from the time of balloon placement through delivery in the Garbose Family Special Delivery Unit.

Weekly prenatal monitoring will occur after the first procedure at the CFDT and planned delivery will occur in the Garbose Family Special Delivery Unit at term.

Postnatal stabilization and subsequent surgery to repair the diaphragm will take place at CHOP.

Infants will be followed at CHOP at 6 months, 12 months, and 2 years of age and then long-term in the Pulmonary Hypoplasia Program at The Children's Hospital of Philadelphia.

• Study Type: Interventional
• Enrollment (Estimated): 25
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Holly L Hedrick, MD; Phone Number: 1-800-468-8376; Email: hedrick@chop.edu
• Study Contact Backup: Name: Sabrina J Flohr, MPH; Email: flohrs@chop.edu

Study Locations

• United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia
      • Contact: Holly L Hedrick, MD; Phone Number: 800-468-8376; Email: hedrick@chop.edu
      • Contact: Sabrina J Flohr, MPH; Phone Number: 1-800-468-8376; Email: flohrs@chop.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Pregnant women age 18 years and older, who are able to consent

2. Singleton pregnancy

   Fetal:

3. Normal Karyotype or chromosomal microarray with non-pathologic variants
4. Diagnosis of Isolated Left CDH with liver up
5. Gestation at enrollment prior to 29 weeks plus 5 days
6. SEVERE pulmonary hypoplasia with Ultrasound Observed/Expected Lung-to-Head Ratio (O/E LHR) < 25%

Exclusion Criteria:

1. Pregnant women < 18 years
2. Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy
3. Technical limitations precluding fetoscopic surgery
4. Rubber latex allergy
5. Preterm labor, cervix shortened (<15 mm at enrollment or within 24 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor, placenta previa
6. Psychosocial ineligibility, precluding consent
7. Fetal Diaphragmatic hernia: right-sided or bilateral, major associated anomalies, isolated left-sided with the O/E LHR ≥ 25%
8. Inability to remain at FETO site during time period of tracheal occlusion, delivery and postnatal care

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: FETO in CDH; 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	Device: GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter; Fetoscopic Endoluminal Tracheal Occlusion (FETO) in CDH with GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Successful balloon placement and removal.	Successful balloon placement and removal will be counted per patient. FETO insertion will be attempted up to 3 times in a single pregnant woman/ fetus. The maximum duration of balloon implantation, if placed at 27 weeks 0 days and removed in the 34th week, is 7 weeks. For those balloons placed later in gestation or removed earlier electively or emergently, the duration will be shorter.	7 weeks

Collaborators and Investigators

• Sponsor: Children's Hospital of Philadelphia
• Investigators: Principal Investigator: Holly L Hedrick, MD, Children's Hospital of Philadelphia and the Center for Fetal Diagnosis and Treatment

Publications and helpful links

General Publications

• 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.
• Deprest J, Nicolaides K, Done' E, Lewi P, Barki G, Largen E, DeKoninck P, Sandaite I, Ville Y, Benachi A, Jani J, Amat-Roldan I, Gratacos E. Technical aspects of fetal endoscopic tracheal occlusion for congenital diaphragmatic hernia. J Pediatr Surg. 2011 Jan;46(1):22-32. doi: 10.1016/j.jpedsurg.2010.10.008.
• 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.
• 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.
• 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.

Helpful Links

• FETO at The Children's Hospital of Philadelphia
• CHOP Center for Fetal Diagnosis and Treatment
• CHOP Pulmonary Hypoplasia Program

Study record dates

Study Major Dates

• Study Start: July 1, 2015
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: August 18, 2015
• First Submitted That Met QC Criteria: September 11, 2015
• First Posted (Estimated): September 15, 2015

Study Record Updates

• Last Update Posted (Estimated): December 18, 2023
• Last Update Submitted That Met QC Criteria: December 12, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Congenital Diaphragmatic Hernia; Tracheal Occlusion
• Additional Relevant MeSH Terms: Congenital Abnormalities; Pathological Conditions, Anatomical; Internal Hernia; Hernia; Hernias, Diaphragmatic, Congenital; Hernia, Diaphragmatic
• Other Study ID Numbers: 15-011714