Testing Safety and Feasibility of Patient-Derived Stem Cells for Treating Congenital Heart Disease in Adults Aged 18 to 40

What this Trial Is About

Researchers are evaluating the safety and feasibility of lab-grown heart cells derived from stem cells for people with congenital heart disease, specifically those with univentricular heart conditions and advanced heart failure. This Phase 1 study aims to answer whether these autologous induced pluripotent stem cells of cardiac lineage iPSC-CL can be safely delivered to humans and if the trials conduct is practical. The study is sponsored by HeartWorks, Inc. and focuses on participants aged 18 to 40 years with severe heart failure and limited treatment options. Participants in the treated group will receive one dose of the investigational iPSC-CL product, with three escalating dose levels assigned based on product availability and treatment timing. Those who enroll but do not receive the product will serve as controls and receive no intervention. The investigational product is personalized, created from the participants own cells, and the study includes a comparison between treated and untreated subjects to assess differences. During the study, participants will undergo testing and monitoring before and after receiving the product, including assessments of heart function and safety markers such as cardiac troponin levels and tumor markers. They must agree to lifelong follow-up to track long-term safety and outcomes. Primary outcomes include short-term safety over 3 months and feasibility over 12 months, with additional monitoring extending up to 15 years. Overall, participant involvement includes ongoing evaluations, laboratory tests, and adherence checks throughout the study period.

Autologous Induced Pluripotent Stem Cells of Cardiac Lineage for Congenital Heart Disease