Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery

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: NCT03431480
Recruitment Status : Completed
First Posted : February 13, 2018
Last Update Posted : July 22, 2022
Sponsor:
Information provided by (Responsible Party):
Salvatore Pepe, Murdoch Childrens Research Institute

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
This study aims to evaluate the safety and feasibility of coronary infusion of autologous placental cord blood mononuclear cells during the Norwood heart operation in newborn hypoplastic left heart syndrome (HLHS) patients.

Condition or disease Intervention/treatment Phase
Hypoplastic Left Heart Syndrome Heart Defects, Congenital Pediatric Disorder Biological: Autologous Human Placental Cord Blood Mononuclear Cells Phase 1

Detailed Description:
This is a Phase I, open label safety study. Autologous cord blood mononuclear cells (stem cell containing, cord blood buffy coat fraction) was delivered by coronary infusion during the Norwood cardiopulmonary bypass operation in 10 antenatally diagnosed HLHS patients within 2-3 days of birth. Safety and clinical status monitoring was performed to Stage II surgical intervention for HLHS (at approximately 3 months of age). Treated patients will continue to be assessed beyond the trial during follow up between Stage II and III (Fontan) surgical interventions.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Safety
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation
Actual Study Start Date : February 16, 2018
Actual Primary Completion Date : November 1, 2021
Actual Study Completion Date : June 30, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Heart Surgery

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: hCBMNC
Autologous human placental cord blood mononuclear cells (buffy coat fraction)
 Biological: Autologous Human Placental Cord Blood Mononuclear Cells
Autologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation
Other Name: Cord blood buffycoat mononuclear cells, incl stem cells


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Incidence of adverse cardiac events [ Time Frame: 1 month ]
    Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke


Secondary Outcome Measures :
  1. Change in right ventricular function -fractional shortening (% units) [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Measured by cardiac imaging with serial echocardiography and MRI scans

  2. Change in right ventricular end-diastolic wall thickness (% units) [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Measured by cardiac imaging with serial echocardiography and MRI scans

  3. Change in right ventricular end-diastolic volume (% units) [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Measured by cardiac imaging with serial echocardiography and MRI scans

  4. Change in right ventricular end-systolic volume (% units) [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Measured by cardiac imaging with serial echocardiography and MRI scans

  5. Increase in body weight [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Body weight measured in kilograms

  6. Composite measure of height and head circumference [ Time Frame: baseline, 1 month, 3 months, 12 months ]
    Body height and head circumference measured in meters


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   2 Days to 4 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and females with antenatally diagnosed Hypoplastic Left Heart Syndrome (all types requiring Norwood operation)
  • Written informed consent by parents/legal guardian
  • Successful aseptic collection of autologous cord blood unit and transfer of buffy coat mononucleocyte fraction to cold storage pending cardiac surgery within 2-4 days

Exclusion Criteria:

Patient:

  • does not have autologous cord blood cells available at the time of cardiopulmonary bypass surgery
  • has evidence of arrhythmia requiring anti-arrhythmia therapy
  • has an additional congenital diagnosis that contributes to conditions such as an immune system disorder, immune deficiency, complex metabolic disorder, brain dysplasia or progressive neurological degenerative disorder
  • has other clinical complexity deemed by the cardiac surgeon to make the patient unsuitable for inclusion in the trial

Mother:

• is serum positive for HIV, hepatitis or other significant pathogen and has known allergies to penicillin, streptomycin or other antibiotic

Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT03431480


Locations
Layout table for location information
Australia, Victoria
Royal Children's Hospital
Melbourne, Victoria, Australia, 3052
Sponsors and Collaborators
Murdoch Childrens Research Institute
Investigators
Layout table for investigator information
Principal Investigator: Salvatore Pepe Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
Principal Investigator: Christian P Brizard Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Salvatore Pepe, Trial Study Coordinator, Murdoch Childrens Research Institute
ClinicalTrials.gov Identifier: NCT03431480    
Other Study ID Numbers: HREC37112A
First Posted: February 13, 2018    Key Record Dates
Last Update Posted: July 22, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: IPD may be available following study publication and discussion between CIs and other researchers regarding future multicentre studies.

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Salvatore Pepe, Murdoch Childrens Research Institute:
cord blood stem cell
Norwood heart surgery
congenital
heart
Additional relevant MeSH terms:
Layout table for MeSH terms
Heart Defects, Congenital
Hypoplastic Left Heart Syndrome
Congenital Abnormalities
Syndrome
Disease
 Pathologic Processes
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases