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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

Tracking Information
First Submitted Date  ICMJE April 27, 2018
First Posted Date  ICMJE May 16, 2018
Last Update Posted Date April 18, 2019
Actual Study Start Date  ICMJE August 1, 2018
Estimated Primary Completion Date November 1, 2027   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 15, 2018)
  • Safety as assessed by vital sign monitoring (heart rate) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by vital sign monitoring (systolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by vital sign monitoring (diastolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by vital sign monitoring (temperature) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by pulmonary status (indicated by peak inspiratory pressure (PIP)) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by pulmonary status (indicated by positive end expiratory pressure (PEEP)) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by pulmonary status (indicated by respiratory rate (RR)) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by pulmonary status (indicated by Fraction of inspired oxygen (FiO2)) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by presence of new infiltrates or altered aeration upon chest radiography [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by cardiovascular status (indicated by heart rate) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by cardiovascular status (indicated by systolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by cardiovascular status (indicated by diastolic blood pressure) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by cardiovascular status (indicated by changes in cardiovascular pharmacologic support) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by infection status (indicated by body temperature) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by infection status (indicated by white blood cell count) [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by infection status (indicated by physical signs of infection) [ Time Frame: daily for 7 days following the initial infusion ]
  • Safety as assessed by liver function (indicated by Alanine aminotransferase (ALT) levels) [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by liver function (indicated by aspartate aminotransferase (AST) levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by liver function (indicated by bilirubin levels) [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by liver function (indicated by albumin levels) [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by blood urea nitrogen (BUN) levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by creatinine levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by carbon dioxide (CO2) levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by glucose levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by serum chloride levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by serum potassium levels [ Time Frame: 7 days following the initial infusion ]
  • Safety as assessed by serum sodium levels [ Time Frame: 7 days following the initial infusion ]
  • 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) ]
  • 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) ]
  • 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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03526588 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 15, 2018)
  • Mortality [ Time Frame: 2 years after birth ]
  • 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) ]
  • 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) ]
  • Duration of extracorporeal membrane oxygenation (ECMO) support [ Time Frame: days from ECMO initiation until decannulation (an average of 3 weeks) ]
  • Duration of ventilatory support [ Time Frame: from initiation of ventilation until extubation (an average of 8 weeks) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
Official Title  ICMJE Umbilical Cord Blood Mononuclear Cells for Hypoxic Neurologic Injury in Infants With Congenital Diaphragmatic Hernia (CDH)
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Congenital Diaphragmatic Hernia
Intervention  ICMJE 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.
Study Arms  ICMJE Experimental: Autologous umbilical cord blood
Intervention: Biological: Autologous umbilical cord blood
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 15, 2018)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 1, 2027
Estimated Primary Completion Date November 1, 2027   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 7 Days   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Matthew T Harting, MD, MS 713-500-7300 matthew.t.harting@uth.tmc.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03526588
Other Study ID Numbers  ICMJE HSC-MS-18-0148
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Matthew Tihen Harting, The University of Texas Health Science Center, Houston
Study Sponsor  ICMJE The University of Texas Health Science Center, Houston
Collaborators  ICMJE Texas Medical Center Regenerative Medicine Consortium
Investigators  ICMJE
Principal Investigator: Matthew T. Harting, MD, MS The University of Texas Health Science Center, Houston
PRS Account The University of Texas Health Science Center, Houston
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP