Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Phase 1
Terminated
- Conditions
- Congenital Diaphragmatic Hernia
- Interventions
- Biological: Autologous umbilical cord blood
- Registration Number
- NCT03526588
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 3
Inclusion Criteria
- 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.
Exclusion Criteria
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Autologous umbilical cord blood Autologous umbilical cord blood -
- Primary Outcome Measures
Name Time Method Safety as assessed by cardiovascular status (indicated by heart rate) daily for 7 days following the initial infusion Safety as assessed by cardiovascular status (indicated by systolic blood pressure) daily for 7 days following the initial infusion Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP)) daily for 7 days following the initial infusion Safety as assessed by vital sign monitoring (heart rate) daily for 7 days following the initial infusion Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP)) daily for 7 days following the initial infusion Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography daily for 7 days following the initial infusion Safety as assessed by liver function (indicated by albumin levels) 7 days following the initial infusion Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels) 7 days following the initial infusion Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels 7 days following the initial infusion Safety as assessed by infection status (indicated by body temperature) daily for 7 days following the initial infusion Safety as assessed by vital sign monitoring (temperature) daily for 7 days following the initial infusion Safety as assessed by vital sign monitoring (systolic blood pressure) daily for 7 days following the initial infusion Safety as assessed by cardiovascular status (indicated by diastolic blood pressure) daily for 7 days following the initial infusion Safety as assessed by blood urea nitrogen (BUN) levels 7 days following the initial infusion Safety as assessed by glucose levels 7 days following the initial infusion Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI) within 14 days of discharge (discharge occurs at about 2-4 months after birth) Safety as assessed by vital sign monitoring (diastolic blood pressure) daily for 7 days following the initial infusion Safety as assessed by pulmonary status (indicated by respiratory rate (RR)) daily for 7 days following the initial infusion Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2)) daily for 7 days following the initial infusion Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support) daily for 7 days following the initial infusion Safety as assessed by infection status (indicated by white blood cell count) 7 days following the initial infusion Safety as assessed by infection status (indicated by physical signs of infection) daily for 7 days following the initial infusion Safety as assessed by liver function (indicated by bilirubin levels) 7 days following the initial infusion Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications at the time of discharge (which is about 2-4 months after birth) Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III) 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 serum chloride levels 7 days following the initial infusion Safety as assessed by creatinine levels 7 days following the initial infusion Safety as assessed by serum potassium levels 7 days following the initial infusion Safety as assessed by carbon dioxide (CO2) levels 7 days following the initial infusion Safety as assessed by serum sodium levels 7 days following the initial infusion
- Secondary Outcome Measures
Name Time Method 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)
Trial Locations
- Locations (1)
The University of Texas Health Science Center at Houston
🇺🇸Houston, Texas, United States