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A plain-language summary of the goals, design and what participants do
This clinical trial is focused on studying a rare genetic disorder called Leukocyte Adhesion Deficiency-I (LAD-I). This condition affects the immune system, making it difficult for the body to fight off infections. The treatment being tested is called LADICell, which is a type of gene therapy. It involves using the patient's own blood stem cells, which are modified in a laboratory to include a new gene called the ITGB2 gene. This gene helps produce a protein known as CD18, which is important for the immune system to function properly.
The purpose of this study is to evaluate the long-term safety and effectiveness of this gene therapy. Participants in the study will receive an infusion of their own modified stem cells. The study will monitor participants over a long period to see how well the treatment works and to check for any side effects. This involves regular follow-up visits where doctors will assess the participants' health and the functioning of their immune system.
Throughout the study, researchers will look for improvements in the symptoms of LAD-I, such as a reduction in infections and skin or oral lesions. They will also monitor for any serious side effects that might occur after the treatment. The study aims to provide valuable information on whether this gene therapy can be a safe and effective treatment option for people with LAD-I.
The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.
4 criteria
1 criterion
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RP-L201 is a type of gene therapy used in this clinical trial. It involves taking a patient's own blood-forming stem cells and modifying them in a laboratory. The modification is done using a special tool called a lentiviral vector, which carries a healthy version of the ITGB2 gene. This gene is important for helping white blood cells function properly. After the modification, the stem cells are infused back into the patient. The goal of this therapy is to help the patient's immune system work better by correcting the genetic problem that causes Leukocyte Adhesion Deficiency-I (LAD-I). This trial is focused on checking how safe and effective this treatment is over a long period of time.
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Germany
Francesourced from the EU Clinical Trials Register and site verification
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