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

This study is currently recruiting participants.
Verified March 2013 by Duke University
Sponsor:
Information provided by (Responsible Party):
Michael Cotten, Duke University Medical Center
ClinicalTrials.gov Identifier:
NCT01445041
First received: September 15, 2011
Last updated: March 15, 2013
Last verified: March 2013
History of Changes
  Purpose

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.

Condition Intervention Phase
Hypoplastic Left Heart Syndrome
 Biological: Autologous Umbilical Cord Blood
 Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Autologous Cord Blood Cells for Patients With HLHS: Phase I Study of Feasibility and Safety

Further study details as provided by Duke University:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Feasibility and preliminary efficacy [ Time Frame: 1 year ] [ Designated as safety issue: No ]

    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.



Estimated Enrollment: 20
Study Start Date: September 2011
Estimated Study Completion Date: September 2015
Estimated Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single infusion of UCB
(autologous red blood cell and volume reduced cord blood cells)
 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.
Experimental: Three infusions of UCB
(autologous red blood cell and volume reduced cord blood cells)
 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.

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.

  Eligibility

Ages Eligible for Study:   up to 2 Days
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01445041

Contacts
Contact: Kimberley Fisher, PhD 919-681-4913 kimberley.fisher@duke.edu

Locations
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27710
Contact: Kimberley A Fisher, PhD     919-681-4913     kimberley.fisher@duke.edu    
Contact: R Drinker         lisa.drinker@duke.edu    
Sub-Investigator: Joanne Kurtzberg, MD            
Sub-Investigator: Ronald N Goldberg, MD            
Sub-Investigator: Ricki Goldstein, MD            
Sub-Investigator: Lisa R Drinker, MD            
Sub-Investigator: Jennifer Li, MD            
Sub-Investigator: Piers CA Barker, MD            
Sub-Investigator: Jon Meliones, MD            
Sub-Investigator: Andrew Lodge, MD            
Sub-Investigator: Robert J Jaquiss, MD            
Sub-Investigator: Kathryn Gustafson, PhD            
Sponsors and Collaborators
Michael Cotten
Investigators
Principal Investigator: Charles M Cotten, MD MHS Duke University
  More Information

Additional Information:
Carolinas Cord Blood Bank web page  This link exits the ClinicalTrials.gov site

Publications:

Responsible Party: Michael Cotten, Associate Professor of Pediatrics, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01445041     History of Changes
Other Study ID Numbers: Pro00024650
Study First Received: September 15, 2011
Last Updated: March 15, 2013
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Duke University:
Hypoplastic Left Heart Syndrome
Autologous Umbilical Cord Blood
Newborn Infants

Additional relevant MeSH terms:
Hypoplastic Left Heart Syndrome
Heart Defects, Congenital
Cardiovascular Abnormalities
 Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities

ClinicalTrials.gov processed this record on May 23, 2013