Autologous Cardiac Stem Cell Injection in Patients With Hypoplastic Left Heart Syndrome: An Open Label Pilot Study.
Overview
- Phase
- Phase 1
- Intervention
- c-kit+ cells
- Conditions
- Hypoplastic Left Heart Syndrome
- Sponsor
- Joshua M Hare
- Enrollment
- 25
- Locations
- 4
- Primary Endpoint
- Number of Incidence of Treatment Related Major Adverse Cardiac Events
- Status
- Completed
- Last Updated
- 3 months ago
Overview
Brief Summary
The objectives of this pilot study are to evaluate the feasibility and safety of intramyocardial injection of autologous c-kit+ cells during the Stage II Bidirectional Cavopulmonary Anastomosis (BDCPA) operation and to observe effects on clinical outcome including right ventricular myocardial function, severity of tricuspid regurgitation, incidence of serious adverse events, re-hospitalizations, changes in health status, the need for transplantation, or mortality.
Investigators
Joshua M Hare
Professor
University of Miami
Eligibility Criteria
Inclusion Criteria
- •For inclusion in the study, subjects must meet all of the inclusion criteria:
- •Subjects with hypoplastic left heart syndrome (HLHS) (all types) requiring Stage I Norwood operation.
Exclusion Criteria
- •Candidates will be excluded from the study if any of the following conditions are met:
- •Subjects undergoing the Stage I Norwood operation who do not have HLHS.
- •Subjects requiring mechanical circulatory support immediately prior to Stage II BDCPA operation (within 5 days).
- •Parent or guardian unwilling or unable to comply with necessary follow-up(s).
- •Mother is serum positive for HIV 1/2, hepatitis B surface antigen or viremic hepatitis C and Treponema pallidum.
- •Subjects who are unsuitable for inclusion in the study in the opinion of the investigator(s).
Arms & Interventions
Open label C-kit+ cells Group A
Group A is an open-label treatment group determining safety and feasibility. Participants enrolled in this group will be receiving previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
Intervention: c-kit+ cells
C-kit+ cells Group B
Participants randomized to Group B Treatment Group will receive previously harvested c-kit+ cells during their Stage II BDCPA operation. Harvested c-kit+ cells will be injected into the right ventricle directly intramyocardially.
Intervention: c-kit+ cells
No Intervention Group
Participants randomized to Group B Control Group will receive only their standard of care (SOC) Stage II BDCPA operation without the injection of harvested c-kit+ cells.
Outcomes
Primary Outcomes
Number of Incidence of Treatment Related Major Adverse Cardiac Events
Time Frame: 30 days
Safety will be reported as the number of incidence of treatment related major adverse cardiac events (MACE). MACE is defined as any of the following: greater than 30 seconds of sustained/symptomatic ventricular tachycardia requiring intervention, cardiogenic shock, unplanned cardiovascular operation due to injection site bleeding, need for new permanent pacemaker, stroke or embolic event to the brain determined by CT scan and death. MACE will be evaluated by the treating physician and assessed using the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.
Number of C-kit+ Products
Time Frame: Day 1
Feasibility will be reported as the number of c-kit+ products that can be manufactured and delivered to subjects
Number of Participants Completing Magnetic Resonance Imaging (MRI)
Time Frame: Baseline, 6 Months, 12 months
Feasibility will be reported as the number of participants that complete the baseline, 6-months, and 12-months follow up MRI.
Change in Right Ventricular Function (RVEF)
Time Frame: Baseline, 6 Months, 12 months
Efficacy will be reported as the change in right ventricular function assessed as a percentage and will be measuring using serial echocardiograms and MRI scan.
Change in Right Ventricular End-diastolic Volume (RVEDV)
Time Frame: Baseline, 6 months, 12 months
Efficacy will be reported as the change in right ventricular end-diastolic assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Change in Right Ventricular End-systolic Volume (RVESV)
Time Frame: Baseline, 6 months, 12 months
Efficacy will be reported as the change in right ventricular end-systolic volume assessed in milliliters per square meter and will be measured using serial echocardiograms and MRI scan.
Change in Tricuspid Regurgitation
Time Frame: Baseline, 6 months, 12 months
Efficacy will be reported as the change tricuspid regurgitation assessed as a percentage and will be measured using serial echocardiograms and MRI scan.
Secondary Outcomes
- Number of Incidence of Serious Adverse Events(Up to 12 months)
- Change in Somatic Growth Velocity - Length (cm)(Baseline, 6 months, 12 months)
- Change in Somatic Growth Velocity - Weight (kg)(Baseline, 6 months, 12 months)
- Change in Somatic Growth Velocity - Head Circumference (cm)(Baseline, 6 months, 12 months)
- Change in Infant Toddler Quality of Life Survey (ITQOL) - Overall Health(Baseline, 12 months)
- Incidence of Mortality or Need for Transplantation(Up to 12 months)