A Pilot Study on the Role of Fetal Endotracheal Occlusion (FETO) in Fetuses With Severe Congenital Diaphragmatic Hernia
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- Eyal Krispin
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Successful Ballon Placement
研究概览
简要总结
The goal of this pilot trial is to learn more about the role of Fetal Endotracheal Occlusion (FETO) as an intervention in fetuses with severe congenital diaphragmatic hernia (CDH). The research team will investigate the feasibility and safety of the FETO procedure, as well as determine whether FETO can improve lung growth before birth, and survival after birth.
This study will enroll 10 pregnant participants to undergo the FETO procedure at a gestational age of 27 weeks 0 days to 29 weeks 6 days. The participant will be monitored for a few weeks, and then the FETO removal procedure will be performed ideally at 34 weeks 0 days to 34 weeks 6 days, but may be indicated earlier as determined by the Maternal Fetal care team. The pregnant participant and their baby will continue to be monitored during delivery and up until the child reaches 2 years of age.
详细描述
The goal of this study is to learn more about the role of Fetal Endotracheal Occlusion (FETO) as an intervention in fetuses with isolated severe congenital diaphragmatic hernia (CDH). CDH is a condition in which the diaphragm fails to completely close, leaving a gap through which abdominal organs can herniate and slide into and out of the chest. In severe cases, abdominal organs move into the chest and stay there, putting pressure on the heart and lungs and potentially causing the disruption or deformation of these structures. Impaired development of the lungs can often lead to a condition known as pulmonary hypertension, a form of high blood pressure that damages the heart. Pulmonary hypoplasia and pulmonary hypertension can be lethal. Congenital diaphragmatic hernia (CDH) affects 1 in 2,200 to 5,000 live births per year.
FETO will be performed with the goal of promoting lung growth and improving neonatal outcomes. FETO is a minimally invasive procedure in which a balloon device is inserted into the trachea of the fetus. The devices involved are the Goldballoon Detachable Balloon (GOLDBAL2) along with the Delivery Microcatheter (BALTACCI-BDPE100). The balloon would be left in place for several weeks and allow the lungs to grow, after which it would be removed, enabling the lungs to mature before birth. Participants will continue to be monitored up until the child reaches 2 years of age in order to assess mental and physical development after FETO.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Pregnant patient 18 and older who is able to consent
- •Singleton pregnancy
- •Ability to reside within 30 minutes of Boston Children's Hospital for the duration of the FETO intervention, from the time of balloon placement until balloon removal
- •Patient has a support person who is able to stay with them for the duration of the pregnancy
- •Reassuring genetic analysis demonstrated by either normal Karyotype, normal fluorescence in situ hybridization (FISH) for chromosomes 13, 18, 21, X and Y, or chromosomal microarray (CMA) with non-pathologic variants
- •Diagnosis of isolated left CDH with liver up
- •Gestation at enrollment prior to 29 weeks 5 days
- •SEVERE pulmonary hypoplasia with ultrasound Observed/Expected Lung-to-Head Ratio (O/E LHR) < 25%
排除标准
- •Maternal contraindication to fetoscopic surgery or severe maternal medical condition in pregnancy
- •Technical limitations precluding fetoscopic surgery, including uterine anomaly such as large or multiple fibroids, or Mullerian duct anomaly
- •Latex allergy
- •Preterm labor, shortened cervix (<20mm at enrollment or within 24 hours of FETO balloon insertion procedure) or uterine anomaly strongly predisposing to preterm labor, placenta previa
- •Severe maternal obesity pre-pregnancy (BMI > 40)
- •Psychosocial ineligibility, precluding consent, as determined by clinic social worker during review
- •Inability to remain at FETO site during time period of tracheal occlusion, delivery, and postnatal care
- •Right-sided or bilateral, left-sided CDH observed-to-expected lung to head ratio > 25% on ultrasound
- •Additional fetal anomaly and chromosomal abnormalities by ultrasound, MRI, or echocardiogram that will significantly worsen prognosis
- •History of incompetent cervix with or without cerclage
- •Placental abnormalities (previa, abruption, accreta) known at time of enrollment
- •Maternal-fetal RH (rhesus) isoimmunization, Kell sensitization, or neonatal alloimmune thrombocytopenia affecting the current pregnancy
- •Maternal HIV, Hepatitis B, Hepatitis C status positive
研究组 & 干预措施
Fetal Endotracheal Occlusion (FETO)
Participants with severe congenital diaphragmatic hernia will undergo the FETO therapy.
干预措施: Fetal Endotracheal Occlusion (FETO) (Device)
结局指标
主要结局
Successful Ballon Placement
时间窗: Gestational age of 27 weeks 0 days to 29 weeks 6 days
Defined as direct visualization of balloon deployment above the carina at the time of FETO procedure
Successful Balloon Removal
时间窗: Prior to delivery, ideally at 34 weeks
Removal of the balloon prior to delivery, ideally during 34 weeks gestation
Balloon placement operative time
时间窗: Gestational age of 27 weeks 0 days to 29 weeks 6 days
Length of FETO procedure for successful balloon placement
Balloon removal operative time
时间窗: Prior to delivery, ideally at 34 weeks
Length of FETO procedure for successful balloon removal
Maternal Complications
时间窗: From balloon placement to delivery
Maternal complications include: preterm labor, premature rupture of membranes, oligohydramnios, polyhydramnios, chorioamnionitis
Type of FETO release
时间窗: Prior to delivery, ideally at 34 weeks
Emergent or non-emergent FETO release
Gestational age at delivery
时间窗: At delivery
Gestational age at delivery will be recorded
次要结局
- Fetal lung volume(Once per week from balloon placement to removal. This measure will be recorded at earliest gestational age of 27 weeks 0 days, and latest at 34+6/7 weeks' gestation.)
- Infant survival(At hospital discharge or 6 months of age, whichever comes first. Hospital discharge typically occurs at 2-3 months of age)
- Presence of neonatal sepsis(From birth until 1 month of age)
- Infant Neurodevelopment(At 6 months, 12 months, and 24 months of age)
- Route of delivery(At delivery)
- Maternal hospitalization(From delivery until discharge, around 2-3 months of age)
- Observed-to-Expected Lung to Head Ratio(Calculated at balloon removal, ideally at 34 weeks gestation)
- Oxygen dependency(At time of discharge, on average at 2-3 months of age)
- NICU (neonatal intensive care unit) stay(From birth until discharge, at an average of 2-3 months of age)
- Presence of periventricular leukomalacia(From birth until 2 months of age)
- Number of infants requiring ECMO (extracorporeal membrane oxygenation)(Birth to 6 months)
- Ventilator support(Up to 2 years of age)
- Presence of intraventricular hemorrhage(Up to 2 years of age)
- Retinopathy of prematurity(From birth to 1 month of age)
- Presence of gastro-esophageal reflux(From birth until 2 years of age)
- CDH repair type(Postnatal, at time of CDH repair, typically within 2 weeks of birth)
- Pulmonary function(At discharge around 2-3 months and at one year of age)
研究者
Eyal Krispin
Interim Director, Fetal Care and Surgery Center
Boston Children's Hospital