Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia

Study Overview
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Brief Summary
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The purpose of this research is to gather information on the safety and effectiveness of a procedure called Fetoscopic Endoluminal Tracheal Occlusion (FETO) at Mayo Clinic. The intent of the FETO procedure is to improve development of the lungs in fetuses diagnosed with severe congenital diaphragmatic hernia (CDH).

Official Title
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Pilot Trial of Fetoscopic Endoluminal Tracheal Occlusion for Severe Left Congenital Diaphragmatic Hernia

Conditions
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Congenital Diaphragmatic Hernia

Intervention / Treatment
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* Device: BALT GOLDBALL 2 detachable latex ballon
* Device: BALTACCIBDPE100 Microcatheter
* Device: Storz fetoscopic operating sheath and miniature telescope/fetoscope


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Singleton pregnancy Normal fetal karyotype or microarray. Results by fluorescence in situ hybridization (FISH) will be acceptable if the patient is > 26 weeks Isolated severe left CDH with O/E LHR < 25% ) Gestation age at enrollment prior to 29 wks plus 6 days. Pulmonary hypoplasia with ultrasound O/E LHR < 25% (measured at 18 0/7 to 29 5/7 weeks) at the time of surgery. 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 Patient meets psychosocial criteria: able to reside within 30 minutes of Mayo Clinic, Rochester and able to comply with the travel for the follow-up requirements of the trial; patient has a support person (e.g. spouse, partner, mother) available to stay with the patient for the duration of the pregnancy at Mayo Clinic Patient is willing and able to give informed consent Appropriate multi-disciplinary counseling performed with maternal-fetal medicine, neonatology, pediatric surgery, genetics, pediatric otolaryngology (ENT) Exclusion Criteria: Multi-fetal pregnancy History of natural rubber latex allergy Preterm labor, cervix shortened (<20 mm) at enrollment or within 24 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor Psychosocial ineligibility, precluding consent: inability to reside within 30 minutes of Mayo Clinic, Rochester and inability to comply with the travel for the follow-up requirements of the trial; patient does not have a support person (e.g. spouse, partner, mother) available to stay with the patient for the duration of the pregnancy at Mayo Clinic Right sided CDH or bilateral CDH, isolated left sided with O/E LHR >25% measured at 18 0/7 to 29 6/7 weeks) as determined by ultrasound Additional fetal anomaly and chromosomal abnormalities by ultrasound, MRI, or echocardiogram that will significantly worsen prognosis. No cases will be removed post hoc if abnormalities are discovered in the course of post-operative monitoring Maternal contraindication to fetoscopic surgery History of incompetent cervix with or without cerclage Placental abnormalities (previa, abruption, accreta) 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. Uterine anomaly such as large or multiple fibroids or mullerian duct abnormality that will make the procedure technically unfeasible No safe or technically feasible fetoscopic approach to balloon placement Participation in another intervention study that influences maternal and fetal morbidity and mortality or participation in this trial in a previous pregnancy

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FETO Group<br>Participants will undergo fetal endoscopic tracheal occlusion (FETO) surgical procedure between 27 weeks 0 days and 29 weeks 6 days gestation. | Device: BALT GOLDBALL 2 detachable latex ballon<br>* Inflated with fluid in the fetal trachea and provide with the occlusion necessary to promote lung growth.<br>Device: BALTACCIBDPE100 Microcatheter<br>* Used to deliver the detachable balloon<br>Device: Storz fetoscopic operating sheath and miniature telescope/fetoscope<br>* Used to percutaneously enter though the maternal abdominal wall, uterus and into the amniotic cavity, fetal mouth, pharynx, larynx, into the trachea where the delivery catheter will be used to deliver the detachable balloon that will provide with the tracheal occlusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Technical success of the balloon placement procedure | The number of successful balloon placement procedures defined as balloon was correctly inflated and placed/secured in the trachea. | Up to 29 weeks gestation | 
| Technical success of balloon retrieval procedure | The number of successful balloon retrieval procedure defined as antenatal removal of the balloon. | Up to 34 weeks gestation | 
| Operative times | FETO placement and release operative times reported in minutes | Up to 34 weeks gestation | 
| Frequency of unplanned balloon removal | The frequency of non-emergent and emergent completion of FETO release (unplanned balloon removal) | Up to 34 weeks gestation | 
| Number of incidences of maternal complications | Maternal complications including preterm labor, premature rupture of membranes, oligohydramnios, polyhydramnios, chorioamnionitis | Up to 41 weeks gestation | 
| Gestational Age at Delivery | Gestation Age reported at time of delivery | Up to 41 weeks gestation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Fetal Lung Growth as measured via Fetal Lung Volume | Fetal Lung Volume as measured via ultrasound | Up to 24 months post partum | 
| Fetal Lung Growth as measured via LHR | Lung area to head circumference Ratio (LHR) as measured via ultrasound | Up to 24 months post partum | 
| Fetal Survival | Survival at 30 days, discharge from the hospital and at 6 months if still hospitalized | Up to 6 months post partum | 
| Fetal Oxygen Dependency | Oxygen dependency graded as none, mild, moderate, severe as assessed by treating physician | Up to 24 months post partum | 
| Occurrence of severe pulmonary hypertension | Number of occurrence of severe pulmonary hypertension in infants based on echocardiogram | Up to 24 months post partum | 
| ECMO Support | Number of infants reported that required Extracorporeal membrane oxygenation (ECMO) support | Up to 24 months post partum | 
| Number of days in NICU | Number of days infant was in neonatal intensive care unit | Up to 24 months post partum | 
| Number of days of ventilator support | Number of reported days infants required ventilator support | Up to 24 months post partum | 
| Number of periventricular leukomalacia at < 2 months postnatally | Number of infants reported presence of periventricular leukomalacia at < 2 months postnatally | Up to 2 months post partum | 
| Number of infant complications | Number of infants reporting the presence of: neonatal sepsis, intraventricular hemorrhage (grade 3 or higher), retinopathy of prematurity (grade 3 or higher) or gastro-esophageal reflux | Up to 24 months post partum | 
| Use of patch or muscle flap | Number of infants reported to require the use of patch or muscle flap | Up to 24 months post partum | 
| Maternal hospitalization | Number of reported days of maternal hospitalization | Up to 24 months post partum | 
| Route of delivery | Number of participants that delivered vaginally and via caesarean section | Day 1 (post partum) |