Chronic granulomatous disease Trials in Disease

Study of G1XCGD Lentiviral Vector in Patients with X-Linked Chronic Granulomatous Disease

This clinical trial is focused on X-linked Chronic Granulomatous Disease, a rare genetic disorder that weakens the immune system and makes it difficult for the body to fight off certain bacterial and fungal infections. The study is testing an innovative gene therapy approach that uses the patient's own blood stem cells.

Who can participate:

• Male patients with confirmed X-linked Chronic Granulomatous Disease, typically older than 23 months (younger patients between 1 and 23 months may be considered with physician approval)
• Patients must have a molecular diagnosis confirmed by DNA sequencing, showing a lack or more than 70% reduction in the activity of the NADPH-oxidase enzyme
• Patients who have experienced at least one severe infection or inflammatory complication that is ongoing, resistant to treatment, or at high risk of returning, and requires hospital care despite regular therapy
• No suitable matched donor should be available after a 3-month search, unless waiting for a match would be too risky
• Patients must not have co-infections with HIV, hepatitis B, or hepatitis C
• Written informed consent from the patient (if an adult) or from a parent or guardian (for children) is required

Who cannot participate:

• Female patients
• Patients outside the specified age range
• Individuals without confirmed X-linked Chronic Granulomatous Disease
• Individuals considered part of vulnerable populations who may have limited ability to give informed consent

What the trial involves:

The treatment uses a special type of cell therapy involving the patient's own blood stem cells, called CD34+ cells. These cells are collected from the patient and then modified in a laboratory using a lentiviral vector called G1XCGD. This vector carries a healthy version of the CYBB gene, which is essential for normal immune function. The modified cells are then given back to the patient through an intravenous injection directly into the bloodstream.

The goal is to help restore the immune system's ability to fight infections effectively. Throughout the study, researchers will monitor patients over a 12-month period to assess safety and effectiveness. This includes checking for any side effects, evaluating improvements in immune function, and monitoring nutritional status, growth, and development.

Investigational treatment:

The study uses autologous CD34+ cells transduced with the G1XCGD lentiviral vector. This gene therapy approach aims to correct the genetic problem that causes the immune system dysfunction in patients with X-linked Chronic Granulomatous Disease. By inserting a functional copy of the necessary gene into the patient's stem cells, the treatment is designed to enable these cells to produce the proteins needed to fight infections effectively.