The purpose of this research is to determine the safety of an experimental drug in human research subjects suffering from angina caused by coronary artery disease who have no other treatment options. 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 the blockage in your coronary arteries and improving blood flow in your heart.
The study drug is designed to seek out cells in your body and deliver a gene that allows those cells to create more of a protein that naturally exists in your body. This process is called gene transfer. The study drug aims to deliver the gene via an injection to the cells of the heart. This will be done in the operating room by an experienced surgeon.
How Does Gene Therapy Work in This Trial?
The gene therapy product XC001 is an altered adenovirus, refered 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.
- Once the vector is delivered to your heart and makes its way into cells, it breaks up while delivering the gene into the cell nucleus, where your DNA is located.
- Your own cell machinery makes VEGF protein and then ships it outside the cell where the protein can help new blood vessels form.
- This is expected to bring more blood and oxygen to areas of your heart that need it.
What To Expect
If you decide to take part in this study, you will come to the clinic for 1 to 3 screening visits to complete some examinations, tests and procedures to find out if you can be in the study.
If you are deemed eligible, you will receive 1 of 4 doses of the experimental drug. You will continue taking all of your other medications prescribed by your doctor. Approximately thirty-three (33) participants will take part in this study. All follow-up visits will be scheduled based off the date you received the experimental drug.
Your participation will last up to 13.5 months. During this period, you will be expected to complete a total of 10 study visits: 3 visits during a 50-day screening period, 1 visit during your overnight stay in the hospital (or more depending on your recovery), and 6 follow-up visits over 12 months after your surgery.
Risks and/or negative effects related to the procedures and experimental drug we are studying could happen; with the administration of the experimental drug and with the surgical procedure; with the study drug itself; and during the tests and procedures used to test effectiveness and safety of the experimental drug.
Before you begin the study, the study doctor will meet with you to explain all the details of the clinical trial. They will answer any and all questions you or your family might have.