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

Heart Defects, Congenital Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03431480
• Other study ID #: HREC37112A
• Status: Completed
• Phase: Phase 1
• Start date: February 16, 2018
• Est. completion date: June 30, 2022

Study information

• Verified date: July 2022
• Source: Murdoch Childrens Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: June 30, 2022
• Est. primary completion date: November 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Days to 4 Days

Eligibility

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

Related Conditions & MeSH terms

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

Intervention

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

Locations

Country	Name	City	State
Australia	Royal Children's Hospital	Melbourne	Victoria

Sponsors (1)

Lead Sponsor	Collaborator
Murdoch Childrens Research Institute

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse cardiac events	Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke	1 month
Secondary	Change in right ventricular function -fractional shortening (% units)	Measured by cardiac imaging with serial echocardiography and MRI scans	baseline, 1 month, 3 months, 12 months
Secondary	Change in right ventricular end-diastolic wall thickness (% units)	Measured by cardiac imaging with serial echocardiography and MRI scans	baseline, 1 month, 3 months, 12 months
Secondary	Change in right ventricular end-diastolic volume (% units)	Measured by cardiac imaging with serial echocardiography and MRI scans	baseline, 1 month, 3 months, 12 months
Secondary	Change in right ventricular end-systolic volume (% units)	Measured by cardiac imaging with serial echocardiography and MRI scans	baseline, 1 month, 3 months, 12 months
Secondary	Increase in body weight	Body weight measured in kilograms	baseline, 1 month, 3 months, 12 months
Secondary	Composite measure of height and head circumference	Body height and head circumference measured in meters	baseline, 1 month, 3 months, 12 months