Fetoscopic Endoluminal Tracheal Occlusion in Severe Left Congenital Diaphragmatic Hernia

Not Applicable

Recruiting

• Conditions: Congenital Diaphragmatic Hernia; Pulmonary Hypoplasia
• Interventions: Device: GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter

• Registration Number: NCT02549820
• Lead Sponsor: Children's Hospital of Philadelphia

Brief Summary

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.

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 Timeline

• Study Start: 2015-07
• Study First Submitted: 2015-08-18
• Study First Submitted QC: 2015-09-11
• Study First Posted: 2015-09-15
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Primary Completion: 2026-02
• Study Completion: 2026-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Female
• Target Recruitment: 25

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
FETO in CDH	GoldBAL2 Detachable Balloon and BALTACCIBDPE100 Delivery Catheter	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

Primary Outcome Measures

Name	Time	Method
Successful balloon placement and removal.	7 weeks	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.

Trial Locations

Locations (1)

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States