Trial of FETO for Severe Congenital Diaphragmatic Hernia

Official title

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

Overview

This is a single site pilot trial to study the feasibility of Fetoscopic Endoluminal Tracheal Occlusion (FETO) therapy in the most severe group of fetuses with congenital diaphragmatic hernia (CDH) at Ann \& Robert H. Lurie Children's Hospital of Chicago (Lurie Children's). This procedure aims to increase fetal lung volume before birth and improve survival after birth. This study will enroll 10 pregnant women who meet study criteria.

Detailed description

A Goldballoon Detachable Balloon (GOLDBAL2) will be inserted into the fetal trachea and deployed with the Delivery Microcatheter (BALTACCI-BDPE100) between 27 weeks 0 days and 29 weeks and 6 days of gestation. The fetal tracheal balloon will be removed between 34 weeks 0 days and 34 weeks 6 days of gestation or earlier as indicated.

After balloon insertion, participants will be followed weekly and must reside within 30 minutes of Lurie Children's until balloon removal. Additionally, participants will have restrictions from return to work, exercise, or intercourse. The children's health status will be followed until two years of age.

Interventions

• Device FETO with Goldballoon Detachable Balloon (GOLDBAL2) along with the delivery Microcatheter (BALTACCI-BDPE100)
  A Goldballoon Detachable Balloon (GOLDBAL2) will be inserted in the airway of the fetus with severe congenital diaphragmatic hernia (o/e LHR \<25% with liver up) during the FETO procedure between gestational age 27 weeks 0 days and 29 weeks and 6 days. The fetal tracheal balloon will be removed at between 34 weeks 0 days and 34 weeks 6 days gestation or earlier as indicated.

Primary outcome measures

• Number of successful placements of Goldballoon Detachable Balloon at gestational age 27 weeks 0 days (27w0d) to 29 weeks 6 days (29w6d) [Time frame: 27 weeks zero days to 29 weeks 6 days]
• Number of successful removals of balloon [Time frame: Removal prior to delivery, ideally at 34 weeks gestation]

Secondary outcome measures (9)

• Change in fetal lung growth [Time frame: Baseline (before balloon placement) to first ultrasound after balloon removal]
• Gestational age at delivery [Time frame: At the time of delivery]
• Infant survival [Time frame: Birth to 24 months]
• Number of infants requiring extra corporeal membrane oxygenation (ECMO) support [Time frame: Birth to 6 months]
• Number of infants with sepsis [Time frame: Birth to six months]
• Number of infants with intraventricular hemorrhage (IVH) [Time frame: Birth to six months]
• Length of hospital stay [Time frame: Birth to one year]
• Number of infants requiring supplemental oxygen [Time frame: Birth to 24 months]
• Number of maternal complications [Time frame: Up to 4-6 weeks post-partum]

Eligibility criteria

Inclusion criteria

• Provision of signed and dated informed consent form
• Pregnant individuals age 18 years and older
• Singleton pregnancy
• No pathogenic variants on prenatal chromosomal microarray or pathologic findings on karyotype analysis. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is > 26 weeks
• Isolated left CDH with severe pulmonary hypoplasia with o/e LHR </=25% with liver up (measured at 18 weeks 0 days to 29 weeks 5 days of gestation) OR Isolated right CDH with severe pulmonary hypoplasia with o/e LHR </=35% with liver up (measured at 18 weeks 0 days to 29 weeks 5 days of gestation)
• Gestational age at FETO procedure 27 weeks 0 days to 29 weeks 6 days as determined by clinical information and evaluation of first ultrasound
• Fetal echocardiogram with changes expected with CDH and no major structural cardiac defects
• Stated willingness to comply with all study procedures and lifestyle considerations and availability for the duration of the study

• Willingness to reside within 30 minutes of the Chicago Institute for Fetal Health (CIFH) in the time period between the FETO placement procedure and the balloon retrieval procedure and ability to maintain follow up appointments
• Subject has a support person (e.g., spouse, partner, friend, parent) that is available to stay with her for the duration of the pregnancy near Lurie Children's
• Willingness to comply with study restrictions on work, exercise, and intercourse
• Meets psychosocial criteria

Exclusion criteria

• Rubber latex allergy
• Presence of chromosomal abnormalities or anatomic anomalies that are known to significantly alter survival prognosis (i.e., CDH and congenital heart disease). No cases will be removed post hoc if abnormalities are discovered during post-operative monitoring
• History of preterm labor, cervix shortened to ≤20 mm at enrollment or at 24 hours prior to FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor or placenta previa
• Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy
• History of incompetent cervix with or without cerclage
• Placental abnormalities (previa, abruption, accreta, chorioangioma) 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
• Uterine anomaly such as large or multiple fibroids or mullerian duct abnormality
• There is no safe or technically feasible fetoscopic approach to balloon placement
• Participation in another intervention study that influences maternal and fetal morbidity and mortality
• Any other condition which, in the opinion of the investigator, would compromise safety, feasibility or impede compliance

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 1 center

• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago

Identifiers

NCT: NCT05450653 · 2021-4085

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗