The goal of this clinical trial is to test the safety of lab-grown heart cells made from stem cells in subjects with congenital heart disease. The main questions it aims to answer are:
* Is this product safe to deliver to humans * Is the conduct of this trial feasible
Participants will be asked to:
* Agree to testing and monitoring before and after product administration * Receive investigational product * Agree to lifelong follow-up Researchers will compare subjects from the same pool to see if there is a difference between treated and untreated subjects.
None
Age 18 to 40 years old
Subject must be able to understand and provide informed consent.
Univentricular congenital heart disease.
End-stage systolic heart failure, defined as Class IV according to New York Heart Association (NYHA) with abnormal visually estimated ejection fraction below 40%.
Parallel
iPSC-CL
Biological/VaccineAutologous IPSCL
Short term safety
The primary safety endpoint is short term safety defined as the rate of new or worsening serious adverse events (SAE) from any System Organ Class (SOC) within 3 months of the iPSC-CL delivery as compared to the control arm.
Feasibility
The primary feasibility endpoint is the percentage of individuals with collected skin cells that meet all iPSC-CL release criteria and the percentage of individuals that have cells delivered.
Long term safety
Long term safety measured as new or worsening serious adverse events for two years after iPSC-CL delivery as compared to the control arm.
Cardiac High Sensitivity Troponin T
Change from baseline in Cardiac High Sensitivity Troponin T at 3 hours (±30 min) and 6 hours (±30 min) after iPSC-CL delivery and at 1 month post-surgery as compared to the control arm.
NT-pro-BNP
Change from baseline in NT-pro-BNP levels at 1 and 3 months post iPSC-CL delivery as compared to the control arm.
Tumor marker levels
Change from baseline in tumor marker levels (PSA (males only), CA 125, CEA, CA 19-9, alpha-fetoprotein (AFP), CA 195, Alpha Subunit HCG) 3 months and annually after iPSC-CL delivery as compared to the control arm.
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