Severe Congenital Diaphragmatic Hernia
Conditions
Brief summary
The purpose of this pilot study is to evaluate the feasibility of using Fetoscopic Endoluminal Tracheal Occlusion (FETO) in 10 pregnant individuals carrying fetuses with severe congenital diaphragmatic hernia (CDH). FETO surgery will be performed at 27 weeks - 29 weeks and 6 days of gestation, and the fetoscopic removal of the balloon will be performed at 34 weeks - 34 weeks and 6 days of gestation. The primary endpoint is successful placement and removal of the balloon.
Interventions
DEVICEGoldballoon Detachable Balloon (GOLDBAL2)
GOLDBAL2 will be inserted into the fetal trachea and deployed with the Delivery Microcatheter (BALTACCI-BDPE100).
Sponsors
NYU Langone Health
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Provision of a signed and dated informed consent form 2. Pregnant individuals age 18 years and older 3. Singleton pregnancy 4. A normal karyotype with confirmation by culture results, CMA with non-pathologic variants, whole exome or genome sequencing (WES or WGS). 5. Isolated left CDH with severe pulmonary hypoplasia with o/e LHR less than 25% with liver up (measured at 18 weeks 0 days to 29 weeks 5 days of gestation) 6. Isolated right CDH with severe pulmonary hypoplasia with o/e LHR equal or less than 30% with liver up (measured at 18 weeks 0 days to 29 weeks 5 days of gestation) 7. Gestational age at FETO procedure 27 weeks 0 days to 29 weeks 6 days as determined by clinical information (LMP) and evaluation of first ultrasound 8. Stated willingness to comply with all study procedures and lifestyle considerations and availability for the duration of the study 9. Willing to reside within a 30-minute distance of the NYULH in the time period between the FETO placement procedure and the balloon retrieval procedure, and ability to maintain follow-up appointments 10. The subject has a support person (e.g., spouse, partner, friend, parent) who is available to stay with her for the duration of the pregnancy near NYULH. 11. Willing to comply with restrictions of daily living, including inability to exercise, have intercourse, or return to work 12. Meets psychosocial criteria
Exclusion criteria
1. Natural rubber latex allergy 2. Additional fetal anomaly and chromosomal abnormalities, associated anomalies recognized to alter survival prognosis (i.e., congenital heart disease) or presence of an underlying genetic syndrome (i.e., Fryns) by ultrasound, MRI or echocardiogram at the fetal treatment center. 3. Signs of preterm labor, cervix shortened to ≤20 mm at enrollment or at 24 hours prior to FETO balloon insertion procedure) or a uterine anomaly strongly predisposing to preterm labor. 4. Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy 5. History of incompetent cervix with or without cerclage 6. Placental abnormalities (previa, abruption, accreta, chorioangioma) known at time of enrollment 7. Maternal-fetal Rh isoimmunization, Kell sensitization or neonatal alloimmune thrombocytopenia affecting the current pregnancy 8. 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 9. Uterine anomaly such as large or multiple fibroids or mullerian duct abnormality 10. There is no safe or technically feasible fetoscopic approach to balloon placement 11. Participation in another intervention study that influences maternal and fetal morbidity and mortality 12. Any other condition which, in the opinion of the investigator, would compromise safety, feasibility or impede compliance. 13. Bilateral CDH, isolated left sided CDH with o/e LHR \>/= 25% and isolated right sided CDH \>30% 14. No intrathoracic liver herniation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of successful balloon placements at age 27w0d - 29w6d and removal at 34 weeks of gestation | Up to 35 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Difference between the relative lung volume (o/e LHR and o/e TLV) | Baseline, removal of balloon (up to 35 weeks) | Fetal lung growth will be calculated by the difference between the relative lung volume (observed-to-expected (o/e) lung-to-head ratio (LHR) and o/e total lung volume (TLV)) measured before and after FETO |
Countries
United States
Contacts
CONTACTErin Lonergan, MHA, BSN, RN
CONTACTJose L. Peiro, MD, PhD
PRINCIPAL_INVESTIGATORJose L. Peiro, MD, PhD
NYU Langone Health
Outcome results
None listed