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

Study Status: Suspended
Recruit Status: Suspended
Study Type: Interventional

Official Title: 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 Summary: 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.
Sponsor: Michael Cotten

Current Primary Outcome: 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: Same as current

Current Secondary Outcome: 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: 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.



Information By: Duke University

Dates:
Date Received: September 15, 2011
Date Started: September 2011
Date Completion: December 2017
Last Updated: March 20, 2017
Last Verified: March 2017