Chronic granulomatous disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:25:30 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Chronic granulomatous disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Safety and efficacy of autologous CD34+ cells transduced with a vector encoding human NCF1 in children and adults with p47‑deficient chronic granulomatous disease https://clinicaltrials.eu/trial/safety-and-efficacy-of-autologous-cd34-cells-transduced-with-a-vector-encoding-human-ncf1-in-children-and-adults-with-p47-deficient-chronic-granulomatous-disease/ Fri, 05 Jun 2026 10:55:58 +0000 https://clinicaltrials.eu/trial/safety-and-efficacy-of-autologous-cd34-cells-transduced-with-a-vector-encoding-human-ncf1-in-children-and-adults-with-p47-deficient-chronic-granulomatous-disease/ The study focuses on Chronic granulomatous disease (CGD) caused by a defect called p47phox deficiency. This rare condition makes certain white blood cells unable to produce a chemical called NADPH oxidase activity that helps kill germs, leading to frequent infections and inflammation. The investigational treatment uses the patient’s own blood stem cells, known as CD34+ cells, which are taken out, altered with a lentiviral vector that carries a normal copy of the NCF1 gene, and then returned to the body through an infusion. The product is identified by the code name SGX-001. Before the cells are given back, participants receive a strong chemotherapy regimen, referred to as myeloablative conditioning, to prepare the bone marrow.

The purpose of the study is to evaluate the safety of this single administration. After the infusion, participants are followed for about a year, with regular visits that include physical exams, vital sign checks, blood tests, and heart rhythm recordings (ECG). Doctors will watch for any side effects and will also test the blood to see if at least 10 % of the white blood cells show restored granulocyte function, indicating that the gene therapy is working. The study does not involve a comparison group.

]]> Study of G1XCGD Lentiviral Vector in Patients with X-Linked Chronic Granulomatous Disease https://clinicaltrials.eu/trial/study-of-g1xcgd-lentiviral-vector-in-patients-with-x-linked-chronic-granulomatous-disease/ Wed, 29 Apr 2026 14:27:27 +0000 https://clinicaltrials.eu/trial/study-of-g1xcgd-lentiviral-vector-in-patients-with-x-linked-chronic-granulomatous-disease/ This clinical trial is focused on studying a rare genetic disorder called X-linked Chronic Granulomatous Disease (X-CGD). This condition affects the immune system, making it difficult for the body to fight off certain infections. The study is testing a new treatment involving a special type of cell therapy. The treatment uses the patient’s own blood stem cells, known as CD34+ cells, which are modified in the laboratory with a lentiviral vector called G1XCGD. This vector carries a healthy version of the human CYBB gene, which is important for normal immune function. The modified cells are then injected back into the patient to help restore their immune system.

The purpose of the study is to evaluate the safety and effectiveness of this treatment in patients with X-CGD. Participants will receive the treatment through an intravenous injection, which means it is delivered directly into the bloodstream. The study will monitor the patients over a period of time to see how well the treatment works and to check for any side effects. The main goal is to see if the treatment can help the immune system function better and remain stable over time.

Throughout the study, researchers will look at various aspects of the patients’ health, including their nutritional status, growth, and development. They will also assess how well the immune system is working by checking for improvements in the body’s ability to fight infections. The study aims to provide valuable information on whether this new cell therapy can be a safe and effective option for people with X-CGD.

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