NCT03431480

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2018

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 30, 2018

Completed
14 days until next milestone

First Posted

Study publicly available on registry

February 13, 2018

Completed
3 days until next milestone

Study Start

First participant enrolled

February 16, 2018

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2021

Completed
8 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2022

Completed
Last Updated

July 22, 2022

Status Verified

July 1, 2022

Enrollment Period

3.7 years

First QC Date

January 30, 2018

Last Update Submit

July 19, 2022

Conditions

Hypoplastic Left Heart SyndromeHeart Defects, CongenitalPediatric Disorder

Keywords

cord blood stem cellNorwood heart surgerycongenitalheart

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (6)

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

    baseline, 1 month, 3 months, 12 months

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

    baseline, 1 month, 3 months, 12 months

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

    baseline, 1 month, 3 months, 12 months

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

    baseline, 1 month, 3 months, 12 months

  • Increase in body weight

    baseline, 1 month, 3 months, 12 months

  • +1 more secondary outcomes

Study Arms (1)

hCBMNC

EXPERIMENTAL

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

Biological: Autologous Human Placental Cord Blood Mononuclear Cells

Interventions

Autologous Human Placental Cord Blood Mononuclear CellsBIOLOGICAL

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

Also known as: Cord blood buffycoat mononuclear cells, incl stem cells
hCBMNC

Eligibility Criteria

Age2 Days - 4 Days
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Location

Related Publications (1)

  • Brizard CP, Elwood NJ, Kowalski R, Horton SB, Jones BO, Hutchinson D, Zannino D, Sheridan BJ, Butt W, Cheung MMH, Pepe S. Safety and feasibility of adjunct autologous cord blood stem cell therapy during the Norwood heart operation. J Thorac Cardiovasc Surg. 2023 Dec;166(6):1746-1755. doi: 10.1016/j.jtcvs.2023.07.035. Epub 2023 Jul 30.

    PMID: 37527726DERIVED

MeSH Terms

Conditions

Hypoplastic Left Heart SyndromeHeart Defects, Congenital

Condition Hierarchy (Ancestors)

Cardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • 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)

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: Safety
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Trial Study Coordinator

Study Record Dates

First Submitted

January 30, 2018

First Posted

February 13, 2018

Study Start

February 16, 2018

Primary Completion

November 1, 2021

Study Completion

June 30, 2022

Last Updated

July 22, 2022

Record last verified: 2022-07

Locations

AU(1)