Clinical trial NCT04052828: Fetal Endoscopic Tracheal Occlusion (FETO) Trial for Congenital Diaphragmatic Hernia (CDH)
Official title: Fetal Endoscopic Tracheal Occlusion Trial for Congenital Diaphragmatic Hernia (CDH)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Congenital Diaphragmatic Hernia
Keywords: pregnant
fetal endoscopic tracheal occlusion
Lead sponsor: University of Michigan
Sponsor class: OTHER
First posted: 2019-08-12
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 assess the feasibility and safety of treating severe CDH with Fetal Endoscopic Tracheal Occlusion with the Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100) at Michigan Medicine. The study will enroll pregnant women that meet study criteria. Participants will have placement of FETO between gestational age at 27 weeks plus 0 days and 29 weeks 6 days. The timing for removal of FETO will ideally be between 34 weeks 0 days and 34 weeks and 6 days but ultimately decided by the Fetal Diagnosis and Treatment Center at Michigan Medicine.

This study requires that study participants live within 30 miles of the Von Voigtlander Women's Hospital and C.S. Mott Children's Hospital in order to maintain weekly follow up appointments while the balloon is in place and up to delivery. Additionally, there are lifestyle considerations where participants would be unable to carry on normal daily activities including exercise and sexual intercourse, not be able to work the remainder of the pregnancy, as well as have a support person that is available to stay with such as a spouse, friend, partner, parent.


Eligibility criteria: Inclusion Criteria:

* Signed and dated consent
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Singleton pregnancy
* No pathogenic variants on microarray or pathologic findings on karyotype
* Fetal echocardiogram with changes expected with CDH and no major structural cardiac defects
* Fetal CDH (left or right) with severe pulmonary hypoplasia, defined as observed-to-expected (o/e) lung to head ratios (LHR) \<25% with liver up
* Gestational age at FETO procedure: if o/e LHR \<25% will be done at 27 weeks plus 0 days to 29 weeks plus 6 days
* Meets psychosocial criteria

  * Willing to reside within 30 minutes of Von Voigtlander Women's Hospital and ability to maintain follow up appointments
  * Patient has a support person (e.g. spouse, partner, friend, parent) that is available to stay with her for the duration of the pregnancy near Von Voigtlander Women's Hospital
  * Willing to comply with restrictions of daily living including inability to exercise, have intercourse, or return to work.

Exclusion Criteria:

* Multi-fetal pregnancy
* History of latex allergy
* History of preterm labor or incompetent cervix (requiring cerclage), short cervix (≤20mm), or uterine anomaly predisposing to preterm labor
* Bilateral CDH, unilateral CDH with o/e LHR \> 25%, or unilateral CDH with o/e LHR \<25% but liver completely down in abdomen
* Additional fetal or genetic abnormalities that would impact care after delivery or be known to have an impact on outcome
* Maternal contraindications to elective fetoscopic surgery
* Significant placental abnormalities (abruption, chorioangioma, accreta) known at time of enrollment and/or surgery
* Maternal isoimmunization or neonatal alloimmune thrombocytopenia
* Maternal HIV, Hepatitis B with positive surface antigen, Hepatitis C with presence of virus in maternal blood due to risk of fetal transmission during the procedure
* No safe or feasible fetoscopic approach to balloon placement
* Social work will meet with each patient to evaluate the social situation and support system. Identifiable issues of social instability or compliance with the protocol will exclude her as a potential candidate.

Primary outcomes:
measure: Number of successful placements of the Goldballoon Detachable Balloon (GOLDBAL2) at gestational age 27 weeks zero days (27w0d) to 29 weeks and 6 days (29w6d) gestation.
timeFrame: 27w0d - 29w6d
measure: Number of successful retrievals of the GOLDBAL2
timeFrame: Removal prior to delivery approximately 34 weeks of gestation
Secondary outcomes:
measure: Change in observed to expected total fetal lung to head ratios (o/e TFLV) on prenatal magnetic resonance imaging (MRI)
timeFrame: baseline (prior to balloon placement), approximately 2 weeks after balloon retrieval
measure: Change in fetal lung growth
timeFrame: baseline (before balloon placement), immediately prior to balloon retrieval
measure: Gestational age at delivery
timeFrame: At the time of delivery (up to approximately 34 weeks)
measure: Survival of infant to hospital discharge or 180 days after hospitalized
timeFrame: Discharge from hospital or up to 180 days in the hospital
measure: Number of maternal complications
timeFrame: up to 4-6 weeks postpartum
Other outcomes:
Locations:
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
Study officials:
affiliation: University of Michigan
name: Erin Perrone, MD
role: PRINCIPAL_INVESTIGATOR
References: