A phase I/II, non randomized, monocentric open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-linked chronic granulomatous disease

This study is a clinical trial exploring a new gene therapy for a serious inherited immune condition called X-linked Chronic Granulomatous Disease (X-CGD). People with X-CGD have a faulty gene that stops their immune cells from killing certain germs, leading to severe and recurrent infections and sometimes chronic inflammation. Current treatments help manage the symptoms, but researchers are always looking for better solutions.

The new treatment involves taking some special blood cells rich in stem cells, called CD34+ cells, from the patient's own body. These cells are then sent to a lab where scientists introduce a working copy of the faulty gene into them using a specially designed virus, called a lentiviral vector. This virus acts like a delivery truck, carrying the healthy gene into the cells. Once the cells have the working gene, they are given back to the patient. The hope is that these corrected cells will then produce healthy immune cells, helping the body to fight off infections more effectively.

The main goals of this study are to carefully check the safety of this new treatment and to see if it helps improve the patient's immune system. Researchers will be looking for any side effects and will also measure if the patient's immune cells start working properly again at least a year after the treatment. They will also observe if the treatment improves overall health, helps clear existing infections, and how long the corrected cells last in the body.