The EXACT clinical trial is a Phase 1/2 multicenter, open-label, single arm, dose escalation trial. Approximately 12 subjects (N=3 per cohort) who have refractory angina will be enrolled into 4 ascending dose groups, followed by an expansion of the highest tolerated dose with 21 additional subjects.
The trial is designed to test the safety and tolerability of an investigational product called XC001. This is the first research study in which this drug will be administered to humans. The study drug is designed to promote new blood vessels in the heart, with these new blood vessels bypassing diseased blood vessels and improving blood flow in the heart.
The study drug is designed to deliver a gene that allows targeted heart cells to create more of a protein that already naturally exists. This process is called gene transfer. The study drug aims to deliver the gene to cells via an injection to the heart. This will be done during a one-time administration of the investigational product XC001 directly to the muscle tissue of the heart by an experienced surgeon.
Participation includes 9 clinic visits during the first 6 months and then a final visit 1 year after receiving the study drug for a total duration of approximately 13 months.
The gene therapy product XC001 is an altered adenovirus, referred to as the vector, that includes a gene for human vascular endothelial growth factor or VEGF. The vector cannot reproduce and has been shown to be safe in human studies.