Clinical trial NCT05450653: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Congenital Diaphragmatic Hernia
Keywords: Hernia, Diaphragmatic, Congenital
Fetal Endoscopic Tracheal Occlusion
Fetoscopic Endoluminal Tracheal Occlusion
Pregnant
Humans
Fetoscopy
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2022-07-11
Enrollment: 10
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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 or right 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)
* 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

Primary outcomes:
measure: Number of successful placements of Goldballoon Detachable Balloon at gestational age 27 weeks 0 days (27w0d) to 29 weeks 6 days (29w6d)
timeFrame: 27 weeks zero days to 29 weeks 6 days
measure: Number of successful removals of balloon
timeFrame: Removal prior to delivery, ideally at 34 weeks gestation
Secondary outcomes:
measure: Change in fetal lung growth
timeFrame: Baseline (before balloon placement) to first ultrasound after balloon removal
measure: Gestational age at delivery
timeFrame: At the time of delivery
measure: Infant survival
timeFrame: Birth to 24 months
measure: Number of infants requiring extra corporeal membrane oxygenation (ECMO) support
timeFrame: Birth to 6 months
measure: Number of infants with sepsis
timeFrame: Birth to six months
measure: Number of infants with intraventricular hemorrhage (IVH)
timeFrame: Birth to six months
measure: Length of hospital stay
timeFrame: Birth to one year
measure: Number of infants requiring supplemental oxygen
timeFrame: Birth to 24 months
measure: Number of maternal complications
timeFrame: Up to 4-6 weeks post-partum
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann & Robert H Lurie Children's Hospital of Chicago
name: Aimen Shaaban, MD
role: PRINCIPAL_INVESTIGATOR
References: