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

Primary Purpose

Hypoplastic Left Heart Syndrome, Heart Defects, Congenital, Pediatric Disorder

Status

Completed

Phase

Phase 1

Locations

Australia

Study Type

Interventional

Intervention

Autologous Human Placental Cord Blood Mononuclear Cells

About this trial

This is an interventional treatment trial for Hypoplastic Left Heart Syndrome focused on measuring cord blood stem cell, Norwood heart surgery, congenital, heart

Eligibility Criteria

2 Days - 4 Days (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Royal Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

hCBMNC

Arm Description

Autologous human placental cord blood mononuclear cells (buffy coat fraction)

Outcomes

Primary Outcome Measures

Incidence of adverse cardiac events

Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke

Secondary Outcome Measures

Change in right ventricular function -fractional shortening (% units)

Measured by cardiac imaging with serial echocardiography and MRI scans

Change in right ventricular end-diastolic wall thickness (% units)

Measured by cardiac imaging with serial echocardiography and MRI scans

Change in right ventricular end-diastolic volume (% units)

Measured by cardiac imaging with serial echocardiography and MRI scans

Change in right ventricular end-systolic volume (% units)

Measured by cardiac imaging with serial echocardiography and MRI scans

Increase in body weight

Body weight measured in kilograms

Composite measure of height and head circumference

Body height and head circumference measured in meters

Full Information

NCT ID

NCT03431480

First Posted

January 30, 2018

Last Updated

July 19, 2022

Sponsor

Murdoch Childrens Research Institute

1. Study Identification

Unique Protocol Identification Number

NCT03431480

Brief Title

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

Official Title

Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation

Study Type

Interventional

2. Study Status

Record Verification Date

July 2022

Overall Recruitment Status

Completed

Study Start Date

February 16, 2018 (Actual)

Primary Completion Date

November 1, 2021 (Actual)

Study Completion Date

June 30, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Murdoch Childrens Research Institute

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hypoplastic Left Heart Syndrome, Heart Defects, Congenital, Pediatric Disorder

Keywords

cord blood stem cell, Norwood heart surgery, congenital, heart

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Model Description

Safety

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

hCBMNC

Arm Type

Experimental

Arm Description

Autologous human placental cord blood mononuclear cells (buffy coat fraction)

Intervention Type

Biological

Intervention Name(s)

Autologous Human Placental Cord Blood Mononuclear Cells

Other Intervention Name(s)

Cord blood buffycoat mononuclear cells, incl stem cells

Intervention Description

Autologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation

Primary Outcome Measure Information:

Title

Incidence of adverse cardiac events

Description

Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke

Time Frame

1 month

Secondary Outcome Measure Information:

Title

Change in right ventricular function -fractional shortening (% units)

Description

Measured by cardiac imaging with serial echocardiography and MRI scans

Time Frame

baseline, 1 month, 3 months, 12 months

Title

Change in right ventricular end-diastolic wall thickness (% units)

Description

Measured by cardiac imaging with serial echocardiography and MRI scans

Time Frame

baseline, 1 month, 3 months, 12 months

Title

Change in right ventricular end-diastolic volume (% units)

Description

Measured by cardiac imaging with serial echocardiography and MRI scans

Time Frame

baseline, 1 month, 3 months, 12 months

Title

Change in right ventricular end-systolic volume (% units)

Description

Measured by cardiac imaging with serial echocardiography and MRI scans

Time Frame

baseline, 1 month, 3 months, 12 months

Title

Increase in body weight

Description

Body weight measured in kilograms

Time Frame

baseline, 1 month, 3 months, 12 months

Title

Composite measure of height and head circumference

Description

Body height and head circumference measured in meters

Time Frame

baseline, 1 month, 3 months, 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Days

Maximum Age & Unit of Time

4 Days

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Salvatore Pepe

Organizational Affiliation

Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Christian P Brizard

Organizational Affiliation

Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)

Official's Role

Principal Investigator

Facility Information:

Facility Name

Royal Children's Hospital

City

Melbourne

State/Province

Victoria

ZIP/Postal Code

3052

Country

Australia

12. IPD Sharing Statement

Plan to Share IPD

Undecided

IPD Sharing Plan Description

IPD may be available following study publication and discussion between CIs and other researchers regarding future multicentre studies.

Learn more about this trial

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

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