Safety and Feasibility of Autologous Induced Pluripotent Stem Cells of Cardiac Lineage in Subjects With Congenital Heart Disease
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.
∙ Individuals who meet all the following criteria are eligible for enrollment as study participants:
• 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%.
• Prognosis of 1 to 1.5 years survival at time of skin biopsy.
• The patient falls into one of the following categories:
‣ Currently listed for heart transplantation at an accredited program in the US but has an expected waiting time for a suitable organ that is likely longer than anticipated life-expectancy.
⁃ Has been denied access to a heart transplantation at an accredited US institution.
⁃ Is currently on or planning to be on mechanical support as destination therapy.
• All guideline directed therapy available to the subject has been maximized, for a minimum of 3 months prior to enrollment.
• Adequate social support system that facilitates subject participation in all study required tests and procedures and supports the subject's ability to comply with long-term study requirements.