Hypoplastic Left Heart SyndromePhase 1NCT03431480
Safety of Autologous Cord Blood Cells in HLHS Patients During Norwood Heart Surgery
A Phase 1 study testing Autologous Human Placental Cord Blood Mononuclear Cells in people with Hypoplastic Left Heart Syndrome and Heart Defects, Congenital, run by Murdoch Childrens Research Institute at 1 site in Australia.
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. Read more about this study
- Who is it for?
- Ages 2 to 4 days
- Conditions the study is for
- Hypoplastic Left Heart Syndrome, Heart Defects, Congenital, Pediatric Disorder
- What is being tested?
- Autologous Human Placental Cord Blood Mononuclear Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteAustralia
- Study dates
- 16 Feb 2018to 30 Jun 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 4 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation
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.
Read the 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.
- Being tested
- Autologous Human Placental Cord Blood Mononuclear Cells
- Keywords
- cord blood stem cell, Norwood heart surgery, congenital, heart
- Medical subject headings
- Hypoplastic Left Heart Syndrome, Heart Defects, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Human Placental Cord Blood Mononuclear CellsBiological
Also called Cord blood buffycoat mononuclear cells, incl stem cells
Autologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation
| Study group | Role | What they receive |
|---|---|---|
| hCBMNC | Experimental | Autologous human placental cord blood mononuclear cells (buffy coat fraction) Receives: Biological: Autologous Human Placental Cord Blood Mononuclear Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse cardiac events
Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke
Measured: 1 month
Secondary outcomes (6)
Change in right ventricular function -fractional shortening (% units)
Measured by cardiac imaging with serial echocardiography and MRI scans
Measured: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-diastolic wall thickness (% units)
Measured by cardiac imaging with serial echocardiography and MRI scans
Measured: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-diastolic volume (% units)
Measured by cardiac imaging with serial echocardiography and MRI scans
Measured: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-systolic volume (% units)
Measured by cardiac imaging with serial echocardiography and MRI scans
Measured: baseline, 1 month, 3 months, 12 months
Increase in body weight
Body weight measured in kilograms
Measured: baseline, 1 month, 3 months, 12 months
Composite measure of height and head circumference
Body height and head circumference measured in meters
Measured: baseline, 1 month, 3 months, 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2018
- Study start16 Feb 2018
- Primary completion1 Nov 2021
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hypoplastic Left Heart SyndromeStudies of the same condition, open and under way.
- More trials run by Murdoch Childrens Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Safety
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Murdoch Childrens Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Salvatore Pepe, Murdoch Childrens Research Institute
- Investigators
- Salvatore Pepe, Principal investigator, Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)Christian P Brizard, Principal investigator, Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03431480
- Sponsor's protocol ID
- HREC37112A
- Status last verified
- Jul 2022
- First submitted
- 30 Jan 2018
- Last updated
- 22 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: IPD may be available following study publication and discussion between CIs and other researchers regarding future multicentre studies.
Publications
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.
PubMed 37527726
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Study of Autologous Cord Blood Stem Cell Treatment in Hypoplastic Left Heart Syndrome Patients Undergoing the Norwood Heart Operation
- ClinicalTrials.gov ID
- NCT03431480
- Acronym / sponsor ID
- HREC37112A
- Keywords
- cord blood stem cell, Norwood heart surgery, congenital, heart
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Murdoch Childrens Research InstituteUniversity, hospital or other
- Study start
- 16 Feb 2018 (actual)
- Primary completion
- 1 Nov 2021 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 13 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Safety
| Arm | Type | Receives |
|---|---|---|
| hCBMNC | Experimental | Biological: Autologous Human Placental Cord Blood Mononuclear CellsAutologous human placental cord blood mononuclear cells (buffy coat fraction) |
Who may qualify
- Ages
- 2 to 4 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (7 listed)
- 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
- is serum positive for HIV, hepatitis or other significant pathogen and has known allergies to penicillin, streptomycin or other antibiotic
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Human Placental Cord Blood Mononuclear CellsAlso: Cord blood buffycoat mononuclear cells, incl stem cellsAutologous human placental cord blood mononuclear cells are infused into the coronary artery during the Norwood heart operation | Biological | hCBMNC |
Primary outcome
Incidence of adverse cardiac events
Time frame: 1 month
Monitoring of adverse events including death, heart or other organ failure, myocardial infarction, sustained/symptomatic ventricular tachycardia, bleeding, stroke
6 secondary outcomes
Change in right ventricular function -fractional shortening (% units)
Time frame: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-diastolic wall thickness (% units)
Time frame: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-diastolic volume (% units)
Time frame: baseline, 1 month, 3 months, 12 months
Change in right ventricular end-systolic volume (% units)
Time frame: baseline, 1 month, 3 months, 12 months
Increase in body weight
Time frame: baseline, 1 month, 3 months, 12 months
Composite measure of height and head circumference
Time frame: baseline, 1 month, 3 months, 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2022.
- Derived from this study: PubMed 37527726Brizard 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.
- Individual participant dataSharing planned: Undecided. IPD may be available following study publication and discussion between CIs and other researchers regarding future multicentre studies.
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Prenatal Maternal Mental Health and Neurodevelopment in Congenital Heart Disease
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Active, not recruitingObservational study
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Salvatore Pepe principal investigator
Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
Christian P Brizard principal investigator
Melbourne Children's Campus (incorporating The Royal Children's Hospital, Murdoch Children's Research Institute, the University of Melbourne Department of Paediatrics)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Human Placental Cord Blood Mononuclear Cells in people with Hypoplastic Left Heart Syndrome and Heart Defects, Congenital, run by Murdoch Childrens Research Institute at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse cardiac events (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03431480, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2022.