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

Sponsor

Murdoch Childrens Research Institute (Other)

Overall Status

Completed

CT.gov ID

NCT03431480

Collaborator

(none)

Enrollment

Location

Arm

52.4

Actual Duration (Months)

0.2

Patients Per Site Per Month

Study Details

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.

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

Study Type:

Interventional

Actual Enrollment :

10 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

SafetySafety

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 :

Feb 16, 2018

Actual Primary Completion Date :

Nov 1, 2021

Actual Study Completion Date :

Jun 30, 2022

Arms and Interventions

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 Names: Cord blood buffycoat mononuclear cells, incl stem cells

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 Names:

Cord blood buffycoat mononuclear cells, incl stem cells

Outcome Measures

Primary Outcome Measures

Incidence of adverse cardiac events [1 month]
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) [baseline, 1 month, 3 months, 12 months]
Measured by cardiac imaging with serial echocardiography and MRI scans
Change in right ventricular end-diastolic wall thickness (% units) [baseline, 1 month, 3 months, 12 months]
Measured by cardiac imaging with serial echocardiography and MRI scans
Change in right ventricular end-diastolic volume (% units) [baseline, 1 month, 3 months, 12 months]
Measured by cardiac imaging with serial echocardiography and MRI scans
Change in right ventricular end-systolic volume (% units) [baseline, 1 month, 3 months, 12 months]
Measured by cardiac imaging with serial echocardiography and MRI scans
Increase in body weight [baseline, 1 month, 3 months, 12 months]
Body weight measured in kilograms
Composite measure of height and head circumference [baseline, 1 month, 3 months, 12 months]
Body height and head circumference measured in meters

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Days to 4 Days

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Royal Children's Hospital	Melbourne	Victoria	Australia	3052

Sponsors and Collaborators

Murdoch Childrens Research Institute

Investigators

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)