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Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)

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ClinicalTrials.gov Identifier: NCT03526588
Recruitment Status : Recruiting
First Posted : May 16, 2018
Last Update Posted : April 18, 2019
Sponsor:
Collaborator:
Texas Medical Center Regenerative Medicine Consortium
Information provided by (Responsible Party):
Matthew Tihen Harting, The University of Texas Health Science Center, Houston

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).

Condition or disease Intervention/treatment Phase
Congenital Diaphragmatic Hernia Biological: Autologous umbilical cord blood Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Actual Study Start Date : August 1, 2018
Estimated Primary Completion Date : November 1, 2027
Estimated Study Completion Date : November 1, 2027


Arm Intervention/treatment
Experimental: Autologous umbilical cord blood Biological: Autologous umbilical cord blood
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 Measures :
  1. Safety as assessed by vital sign monitoring (heart rate) [ Time Frame: daily for 7 days following the initial infusion ]
  2. Safety as assessed by vital sign monitoring (systolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  3. Safety as assessed by vital sign monitoring (diastolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  4. Safety as assessed by vital sign monitoring (temperature) [ Time Frame: daily for 7 days following the initial infusion ]
  5. Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP)) [ Time Frame: daily for 7 days following the initial infusion ]
  6. Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP)) [ Time Frame: daily for 7 days following the initial infusion ]
  7. Safety as assessed by pulmonary status (indicated by respiratory rate (RR)) [ Time Frame: daily for 7 days following the initial infusion ]
  8. Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2)) [ Time Frame: daily for 7 days following the initial infusion ]
  9. Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography [ Time Frame: daily for 7 days following the initial infusion ]
  10. Safety as assessed by cardiovascular status (indicated by heart rate) [ Time Frame: daily for 7 days following the initial infusion ]
  11. Safety as assessed by cardiovascular status (indicated by systolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  12. Safety as assessed by cardiovascular status (indicated by diastolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  13. Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support) [ Time Frame: daily for 7 days following the initial infusion ]
  14. Safety as assessed by infection status (indicated by body temperature) [ Time Frame: daily for 7 days following the initial infusion ]
  15. Safety as assessed by infection status (indicated by white blood cell count) [ Time Frame: 7 days following the initial infusion ]
  16. Safety as assessed by infection status (indicated by physical signs of infection) [ Time Frame: daily for 7 days following the initial infusion ]
  17. Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels) [ Time Frame: 7 days following the initial infusion ]
  18. Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels [ Time Frame: 7 days following the initial infusion ]
  19. Safety as assessed by liver function (indicated by bilirubin levels) [ Time Frame: 7 days following the initial infusion ]
  20. Safety as assessed by liver function (indicated by albumin levels) [ Time Frame: 7 days following the initial infusion ]
  21. Safety as assessed by blood urea nitrogen (BUN) levels [ Time Frame: 7 days following the initial infusion ]
  22. Safety as assessed by creatinine levels [ Time Frame: 7 days following the initial infusion ]
  23. Safety as assessed by carbon dioxide (CO2) levels [ Time Frame: 7 days following the initial infusion ]
  24. Safety as assessed by glucose levels [ Time Frame: 7 days following the initial infusion ]
  25. Safety as assessed by serum chloride levels [ Time Frame: 7 days following the initial infusion ]
  26. Safety as assessed by serum potassium levels [ Time Frame: 7 days following the initial infusion ]
  27. Safety as assessed by serum sodium levels [ Time Frame: 7 days following the initial infusion ]
  28. 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) ]
  29. 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) ]
  30. Neurologic/neurodevelopmental status as assessed by Bayley Scales of Infant and Toddler Development-III (BSID-III) [ Time Frame: 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.


Secondary Outcome Measures :
  1. Mortality [ Time Frame: 2 years after birth ]
  2. 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) ]
  3. 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) ]
  4. Duration of extracorporeal membrane oxygenation (ECMO) support [ Time Frame: days from ECMO initiation until decannulation (an average of 3 weeks) ]
  5. Duration of ventilatory support [ Time Frame: from initiation of ventilation until extubation (an average of 8 weeks) ]


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Ages Eligible for Study:   up to 7 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03526588


Contacts
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Contact: Matthew T Harting, MD, MS 713-500-7300 matthew.t.harting@uth.tmc.edu

Locations
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United States, Texas
The University of Texas Health Science Center at Houston Recruiting
Houston, Texas, United States, 77030
Contact: Matthew T. Harting, MD, MS    855-566-6273      
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Texas Medical Center Regenerative Medicine Consortium
Investigators
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Principal Investigator: Matthew T. Harting, MD, MS The University of Texas Health Science Center, Houston

Additional Information:
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Responsible Party: Matthew Tihen Harting, Assistant Professor, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT03526588     History of Changes
Other Study ID Numbers: HSC-MS-18-0148
First Posted: May 16, 2018    Key Record Dates
Last Update Posted: April 18, 2019
Last Verified: April 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Matthew Tihen Harting, The University of Texas Health Science Center, Houston:
CDH
Additional relevant MeSH terms:
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Hernias, Diaphragmatic, Congenital
Hernia
Hernia, Diaphragmatic
Pathological Conditions, Anatomical
Congenital Abnormalities