Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety and Feasibility Study of Umbilical Cord Blood Cells for Infants With Hypoplastic Left Heart Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01445041
Recruitment Status : Terminated (Funding Period Ended)
First Posted : October 3, 2011
Last Update Posted : January 21, 2020
Sponsor:
Information provided by (Responsible Party):
Michael Cotten, Duke University

Tracking Information
First Submitted Date  ICMJE September 15, 2011
First Posted Date  ICMJE October 3, 2011
Last Update Posted Date January 21, 2020
Actual Study Start Date  ICMJE September 1, 2011
Actual Primary Completion Date April 15, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 29, 2011)
Adverse event rates occurring in the pilot study population. The investigators will compare infusion outcomes of infants infused with frozen cells and infants infused with non-frozen cells. [ Time Frame: During Infusions (First 2 months of life) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 15, 2013)
Feasibility and preliminary efficacy [ Time Frame: 1 year ]
Feasibility: volume of cord blood, cell viability, time to prepare/infuse fresh cells and frozen-thawed cells. Preliminary efficacy: neurodevelopmental outcomes at 9 - 12 months, cardiac function as assessed clinically.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 29, 2011)
Feasibility and preliminary efficacy [ Time Frame: 1 year ]
Feasibility: volume of cord blood, cell viablity, time to prepare/infuse fresh cells and frozen-thawed cells. Preliminary efficacy: neurodevelopmental outcomes at 9 - 12 months, cardiac function as assessed clinically.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Feasibility Study of Umbilical Cord Blood Cells for Infants With Hypoplastic Left Heart Syndrome
Official Title  ICMJE Autologous Cord Blood Cells for Patients With HLHS: Phase I Study of Feasibility and Safety
Brief Summary

Further study details as provided by Duke University:

Purpose: To evaluate the feasibility and safety of collecting and infusing autologous umbilical cord blood (UCB) in newborn infants with hypoplastic left heart syndrome (HLHS).

Study Rationale and Hypotheses: The major goal of this study is to determine whether infusion of autologous UCB cells in neonates with hypoplastic left heart syndrome is feasible and safe. The rationale for the study and for the potential benefit of UCB is based upon the following hypotheses:

  1. Infants with HLHS have significant neural injury evidenced from both prenatal and early antenatal brain MRI findings and infusion of UCB cells may lessen neural injury. Although the exact mechanism is unknown, UCB cell infusion may ameliorate neural injury via paracrine and anti-inflammatory effects that enhance post injury repair and may promote endogenous functional compensation of other cortical areas resulting in significant clinical improvements.
  2. UCB cells may also enhance cardiac function, minimize scar formation, and reverse detrimental remodeling after cardiac injury.
Detailed Description The purpose of this pilot study is to evaluate the safety and feasibility of infusions of autologous (the patient's own)umbilical cord blood cells in neonates with hypoplastic left heart syndrome. This is a prospective, randomized Phase I trial designed to assess the safety and feasibility of autologous UCB reinfusion in neonates with Hypoplastic Left Heart Syndrome (HLHS). Neonates who are identified prenatally as having a cardiac lesion consistent with HLHS will be referred to Duke Cardiology for further evaluation. If they meet inclusion criteria, UCB will be collected at the time of delivery and processed (red blood cell- and volume-reduced) for reinfusion. All enrolled infants will receive a dose of fresh UCB cells pre-operatively and ½ of the enrolled infants will be randomly selected to receive a second dose of frozen and thawed UCB cells after stage 1 palliation (5-35 days post-operatively) and a third dose of frozen and thawed UCB cells 2 to 4 weeks after the 2nd infusion. Neurodevelopmental outcome measures will be assessed at 1 month after discharge, at 4-6 months old and 12 months. The results of MRI's and echocardiograms that are obtained per clinical routine will be analyzed and described in study reports.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hypoplastic Left Heart Syndrome
Intervention  ICMJE
  • Biological: Autologous Umbilical Cord Blood
    Infants who meet study enrollment criteria for hypoplastic left heart syndrome in the neonatal period will receive 1 infusion of their own volume reduced cord blood cells. The dose for each infusion is 5x10e7 cells/kg.
  • Biological: Autologous Umbilical Cord Blood
    Infants who meet study enrollment criteria for hypoplastic left heart syndrome in the neonatal period will receive 3 infusions of their own volume reduced cord blood cells. The first infusion will be a fresh, volume-reduced infusion and the subsequent infusions will be thawed and washed infusions. The dose for each infusion is 5x10e7 cells/kg.
Study Arms  ICMJE
  • Experimental: Single infusion of UCB
    (autologous red blood cell and volume reduced cord blood cells)
    Intervention: Biological: Autologous Umbilical Cord Blood
  • Experimental: Three infusions of UCB
    (autologous red blood cell and volume reduced cord blood cells)
    Intervention: Biological: Autologous Umbilical Cord Blood
Publications *

*   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 Terminated
Actual Enrollment  ICMJE
 (submitted: January 16, 2020)
7
Original Estimated Enrollment  ICMJE
 (submitted: September 29, 2011)
20
Actual Study Completion Date  ICMJE April 15, 2016
Actual Primary Completion Date April 15, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Infants > 35 weeks gestational age.
  • Diagnosis: Hypoplastic Left Heart Syndrome.
  • Autologous umbilical cord blood available with a minimum total nucleated cell dose of 1 x 10e7 cells/kg.
  • Parental Consent.

Exclusion Criteria:

  • Chromosomal anomalies identified before the time of infusion.
  • Chromosomal anomalies or congenital anomalies that would prohibit clinicians from initiating surgical repair of the congenital heart defect.
  • Infant is determined by clinical staff to be non-viable and will not receive aggressive care. (No member on the study team will be involved in determining the viability of the neonate.)
  • Autologous umbilical cord blood unit has any of the following:

    • Total nuclear cell count < 1 x 10e7.
    • Positive maternal infectious serology (except CMV).
    • Evidence of infectious contamination of the cord blood unit.
    • Evidence of genetic disease.
  • Unable to obtain parental consent.
  • Mother < 18 years old.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 2 Days   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01445041
Other Study ID Numbers  ICMJE Pro00024650
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Michael Cotten, Duke University
Study Sponsor  ICMJE Michael Cotten
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Charles M Cotten, MD MHS Duke University
PRS Account Duke University
Verification Date January 2020

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