Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Safety as assessed by liver function (indicated by albumin levels)
研究概览
简要总结
The purpose of this study is to investigate the use of autologous umbilical cord blood (UCB) mononuclear cells to mitigate hypoxic neurologic injury among infants with high-risk congenital diaphragmatic hernia (CDH).
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
- 10 Minutes 至 7 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CDH between 20 and 36 weeks estimated gestational age (EGA)
- •Only one of the following fetal criteria and one of the following postnatal criteria must be met for enrollment. Fetal criteria: an ultrasound (US)-obtained observed to expected lung to head ratio (o/e LHR) less than or equal to 35% or 2) a fetal magnetic resonance imaging (fMRI)- obtained observed to expected total fetal lung volume (o/e TFLV) less than or equal to 35%. Postnatal criteria: 1) Cord blood gas (CBG) with potenital hydrogen (pH) <7.0, 2) Arterial blood gas (ABG) with pH <7.2 on 2 gasses within the first 24 hours, 3) Preductal oxygen saturation (O2 sat) <90% x 2 total hours (not necessarily consecutive) within the first 24 hours, or 4) Oxygenation Index (OI) >20 x 2 total hours (not necessarily consecutive) within the first 24 hours.
排除标准
- •Genetic/chromosomal abnormality: Trisomy 21, Trisomy 18, Trisomy 13 or other, significant genetic abnormality. Microdeletions or other mild genetic abnormalities are not considered exclusionary.
- •Severe/major cardiac anomaly: coarctation of the aorta, combined atrial and ventricular septal defects, hypoplastic left heart syndrome, tetralogy of fallot, double outlet right ventricle, atrioventricular canal defects, or other hemodynamically significant defects.
- •Moderate/severe neurologic / intracranial abnormality: Grade III or IV intraparenchymal hemorrhage, space occupying mass or lesion, or clinically significant traumatic lesion such as a subdural or epidural hemorrhage.
- •Prematurity <30 weeks estimated gestational age (EGA): Birth at 29 6/7 weeks or before
- •Participation in an alternative prenatal intervention study: Fetoscopic Endotracheal Occlusion (FETO)
- •Unwillingness / inability to return for follow-up evaluation and assessment
结局指标
主要结局
Safety as assessed by liver function (indicated by albumin levels)
时间窗: 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by heart rate)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by systolic blood pressure)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP))
时间窗: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (heart rate)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP))
时间窗: daily for 7 days following the initial infusion
Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography
时间窗: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by body temperature)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (temperature)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by vital sign monitoring (systolic blood pressure)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by diastolic blood pressure)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels)
时间窗: 7 days following the initial infusion
Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels
时间窗: 7 days following the initial infusion
Safety as assessed by blood urea nitrogen (BUN) levels
时间窗: 7 days following the initial infusion
Safety as assessed by glucose levels
时间窗: 7 days following the initial infusion
Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI)
时间窗: within 14 days of discharge (discharge occurs at about 2-4 months after birth)
Safety as assessed by vital sign monitoring (diastolic blood pressure)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by respiratory rate (RR))
时间窗: daily for 7 days following the initial infusion
Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2))
时间窗: daily for 7 days following the initial infusion
Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by infection status (indicated by white blood cell count)
时间窗: 7 days following the initial infusion
Safety as assessed by infection status (indicated by physical signs of infection)
时间窗: daily for 7 days following the initial infusion
Safety as assessed by liver function (indicated by bilirubin levels)
时间窗: 7 days following the initial infusion
Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications
时间窗: at the time of discharge (which is about 2-4 months after birth)
Safety as assessed by serum chloride levels
时间窗: 7 days following the initial infusion
Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III)
时间窗: 2 years after birth
The Bayley-III is an individually-administered examination that assesses the current developmental functioning of infants and young children from birth to 42 months of age. The Bayley is a standardized, norm-referenced measure that assesses development in Cognitive, Language and Motor domains. Composite standard scores can be derived that have a mean of 100 and a standard deviation of 15.
Safety as assessed by creatinine levels
时间窗: 7 days following the initial infusion
Safety as assessed by serum potassium levels
时间窗: 7 days following the initial infusion
Safety as assessed by carbon dioxide (CO2) levels
时间窗: 7 days following the initial infusion
Safety as assessed by serum sodium levels
时间窗: 7 days following the initial infusion
次要结局
- Duration of extracorporeal membrane oxygenation (ECMO) support(days from ECMO initiation until decannulation (an average of 3 weeks))
- Duration of ventilatory support(from initiation of ventilation until extubation (an average of 8 weeks))
- Mortality(2 years after birth)
- Length of stay in hospital(from birth to discharge or death, whichever occurs first (discharge occurs at about 2-4 months after birth))
- Progression of pulmonary hypertension as assessed by echocardiography(within 24 hours of birth, prior to operative repair (occurs between day 2 & 14 of life), prior to discharge (usually 2-6 months), and after discharge (2wks-6 months following discharge))
研究者
Matthew T Harting
Assistant Professor
The University of Texas Health Science Center, Houston