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

This study has suspended participant recruitment.
(Pending Additional Funding)
Sponsor:
ClinicalTrials.gov Identifier:
NCT01445041
First Posted: October 3, 2011
Last Update Posted: March 21, 2017
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.
Information provided by (Responsible Party):
Michael Cotten, Duke University Medical Center
  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
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Autologous Cord Blood Cells for Patients With HLHS: Phase I Study of Feasibility and Safety

Resource links provided by NLM:


Further study details as provided by Michael Cotten, Duke University Medical Center:

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) ]

Secondary Outcome Measures:
  • 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.



Estimated Enrollment: 20
Study Start Date: September 2011
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2017 (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

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 2 Days   (Child)
Sexes Eligible for Study:   All
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
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): NCT01445041


Locations
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Michael Cotten
Investigators
Principal Investigator: Charles M Cotten, MD MHS Duke University
  More Information

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
First Submitted: September 15, 2011
First Posted: October 3, 2011
Last Update Posted: March 21, 2017
Last Verified: March 2017

Keywords provided by Michael Cotten, Duke University Medical Center:
Hypoplastic Left Heart Syndrome
Autologous Umbilical Cord Blood
Newborn Infants

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