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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
50
Autologous IPSCL
Mayo Clinic
Rochester, Minnesota, United States
RECRUITINGShort 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.
Time frame: 3 months
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.
Time frame: 12 months
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.
Time frame: 2 years
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.
Time frame: 1 month
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.
Time frame: 3 months
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.
Time frame: Three months from date of treatment and every 12 months after treatment, assessed up to 15 years
Panel Reactive Antibody (PRA) levels
Change from baseline in Panel Reactive Antibody (PRA) levels at 3 and 12 months post-iPSC-CL delivery as compared to the control arm.
Time frame: 12 months
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