Back to resultsUmbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Primary Purpose
Congenital Diaphragmatic Hernia
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Autologous umbilical cord blood
Sponsored by
About this trial
This is an interventional treatment trial for Congenital Diaphragmatic Hernia focused on measuring CDH
Eligibility Criteria
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
Sites / Locations
- The University of Texas Health Science Center at HoustonRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Autologous umbilical cord blood
Arm Description
Outcomes
Primary Outcome Measures
Safety as assessed by vital sign monitoring (heart rate)
Safety as assessed by vital sign monitoring (systolic blood pressure)
Safety as assessed by vital sign monitoring (diastolic blood pressure)
Safety as assessed by vital sign monitoring (temperature)
Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP))
Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP))
Safety as assessed by pulmonary status (indicated by respiratory rate (RR))
Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2))
Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography
Safety as assessed by cardiovascular status (indicated by heart rate)
Safety as assessed by cardiovascular status (indicated by systolic blood pressure)
Safety as assessed by cardiovascular status (indicated by diastolic blood pressure)
Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support)
Safety as assessed by infection status (indicated by body temperature)
Safety as assessed by infection status (indicated by white blood cell count)
Safety as assessed by infection status (indicated by physical signs of infection)
Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels)
Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels
Safety as assessed by liver function (indicated by bilirubin levels)
Safety as assessed by liver function (indicated by albumin levels)
Safety as assessed by blood urea nitrogen (BUN) levels
Safety as assessed by creatinine levels
Safety as assessed by carbon dioxide (CO2) levels
Safety as assessed by glucose levels
Safety as assessed by serum chloride levels
Safety as assessed by serum potassium levels
Safety as assessed by serum sodium levels
Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI)
Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications
Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III)
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.
Secondary Outcome Measures
Mortality
Length of stay in hospital
Progression of pulmonary hypertension as assessed by echocardiography
Duration of extracorporeal membrane oxygenation (ECMO) support
Duration of ventilatory support
Full Information
NCT ID
NCT03526588
First Posted
April 27, 2018
Last Updated
March 31, 2022
Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Texas Medical Center Regenerative Medicine Consortium
1. Study Identification
Unique Protocol Identification Number
NCT03526588
Brief Title
Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Official Title
Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Recruiting
Study Start Date
August 1, 2018 (Actual)
Primary Completion Date
November 1, 2027 (Anticipated)
Study Completion Date
November 1, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
The University of Texas Health Science Center, Houston
Collaborators
Texas Medical Center Regenerative Medicine Consortium
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Diaphragmatic Hernia
Keywords
CDH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Autologous umbilical cord blood
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Autologous umbilical cord blood
Intervention Description
6×10^6 mononuclear cells isolated from the patient's own umbilical cord blood per dose. 4 total doses administered intravenously over 7 days.
Primary Outcome Measure Information:
Title
Safety as assessed by vital sign monitoring (heart rate)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by vital sign monitoring (systolic blood pressure)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by vital sign monitoring (diastolic blood pressure)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by vital sign monitoring (temperature)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP))
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP))
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by pulmonary status (indicated by respiratory rate (RR))
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2))
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by cardiovascular status (indicated by heart rate)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by cardiovascular status (indicated by systolic blood pressure)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by cardiovascular status (indicated by diastolic blood pressure)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by infection status (indicated by body temperature)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by infection status (indicated by white blood cell count)
Time Frame
7 days following the initial infusion
Title
Safety as assessed by infection status (indicated by physical signs of infection)
Time Frame
daily for 7 days following the initial infusion
Title
Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels)
Time Frame
7 days following the initial infusion
Title
Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by liver function (indicated by bilirubin levels)
Time Frame
7 days following the initial infusion
Title
Safety as assessed by liver function (indicated by albumin levels)
Time Frame
7 days following the initial infusion
Title
Safety as assessed by blood urea nitrogen (BUN) levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by creatinine levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by carbon dioxide (CO2) levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by glucose levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by serum chloride levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by serum potassium levels
Time Frame
7 days following the initial infusion
Title
Safety as assessed by serum sodium levels
Time Frame
7 days following the initial infusion
Title
Neurologic/neurodevelopmental status as assessed by intracranial abnormalities upon magnetic resonance imaging (MRI)
Time Frame
within 14 days of discharge (discharge occurs at about 2-4 months after birth)
Title
Neurologic/neurodevelopmental status as assessed by receipt of neurologic pharmacologic medications
Time Frame
at the time of discharge (which is about 2-4 months after birth)
Title
Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III)
Description
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.
Time Frame
2 years after birth
Secondary Outcome Measure Information:
Title
Mortality
Time Frame
2 years after birth
Title
Length of stay in hospital
Time Frame
from birth to discharge or death, whichever occurs first (discharge occurs at about 2-4 months after birth)
Title
Progression of pulmonary hypertension as assessed by echocardiography
Time Frame
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)
Title
Duration of extracorporeal membrane oxygenation (ECMO) support
Time Frame
days from ECMO initiation until decannulation (an average of 3 weeks)
Title
Duration of ventilatory support
Time Frame
from initiation of ventilation until extubation (an average of 8 weeks)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Minutes
Maximum Age & Unit of Time
7 Days
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Matthew T Harting, MD, MS
Phone
713-500-7300
Email
matthew.t.harting@uth.tmc.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Matthew T. Harting, MD, MS
Organizational Affiliation
The University of Texas Health Science Center, Houston
Official's Role
Principal Investigator
Facility Information:
Facility Name
The University of Texas Health Science Center at Houston
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Matthew T. Harting, MD, MS
Phone
855-566-6273
12. IPD Sharing Statement
Links:
URL
http://childrens.memorialhermann.org/conditions/cdh-treatment/
Description
The Comprehensive Center for CDH Care at Children's Memorial Hermann Hospital and The University of Texas Health Science Center in Houston (Texas Medical Center)
Learn more about this trial
Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
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