Clinical trial NCT03431480: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hypoplastic Left Heart Syndrome
Heart Defects, Congenital
Pediatric Disorder
Keywords: cord blood stem cell
Norwood heart surgery
congenital
heart
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
First posted: 2018-02-13
Enrollment: 10
Sex: ALL
Minimum age (years): 2
Maximum age (years): 4
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Safety
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Incidence of adverse cardiac events
timeFrame: 1 month
Secondary outcomes:
measure: Change in right ventricular function -fractional shortening (% units)
timeFrame: baseline, 1 month, 3 months, 12 months
measure: Change in right ventricular end-diastolic wall thickness (% units)
timeFrame: baseline, 1 month, 3 months, 12 months
measure: Change in right ventricular end-diastolic volume (% units)
timeFrame: baseline, 1 month, 3 months, 12 months
measure: Change in right ventricular end-systolic volume (% units)
timeFrame: baseline, 1 month, 3 months, 12 months
measure: Increase in body weight
timeFrame: baseline, 1 month, 3 months, 12 months
measure: Composite measure of height and head circumference
timeFrame: baseline, 1 month, 3 months, 12 months
Other outcomes:
Locations:
city: Melbourne
country: Australia
facility: Royal Children's Hospital
state: Victoria
Study officials:
affiliation: Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
name: Salvatore Pepe
role: PRINCIPAL_INVESTIGATOR
affiliation: Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
name: Christian P Brizard
role: PRINCIPAL_INVESTIGATOR
References: