In short
Currently, there is one ongoing clinical trial focused on gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I). This trial is evaluating the long-term safety and effectiveness of a treatment that uses the patient's own modified stem cells to help the immune system function properly. (Also known as: LAD-I)
Clinical trial locations
Study on Long-Term Safety and Efficacy of Gene Therapy for Leukocyte Adhesion Deficiency-I Using RP-L201 in Patients with LAD-I
This clinical trial is taking place in Spain and focuses on a rare genetic disorder called Leukocyte Adhesion Deficiency-I, which affects the immune system and makes it difficult for the body to fight off infections. The treatment being tested is called LADICell, which is a type of gene therapy that uses the patient's own blood stem cells.
Main inclusion criteria: To participate in this study, patients must have been enrolled in an earlier Phase I/II study called RP-L201-0318 and must have already received the investigational treatment RP-L201 in that previous study. Participants need to be willing and able to follow the study visit schedule and meet all study requirements. They must also provide written informed consent to participate in the trial.
Main exclusion criteria: There are no specific exclusion criteria listed for this trial, meaning that if patients meet the inclusion requirements, they are eligible to participate.
Focus and goal of the trial: The main purpose of this study is to evaluate the long-term safety and effectiveness of gene therapy using RP-L201 in patients who have already received this treatment. The therapy involves modifying the patient's own stem cells in a laboratory to include a new gene called the ITGB2 gene, which helps produce a protein known as CD18. This protein is essential for the immune system to function properly. Participants will receive an infusion of their own modified stem cells and will be monitored over an extended period, potentially until April 2037, to assess how well the treatment works and to check for any side effects. Doctors will look for improvements in symptoms such as a reduction in infections and skin or oral lesions, and will also monitor for any serious side effects that might occur after treatment.
Investigational drug: The trial uses RP-L201, which is a gene therapy that takes a patient's own blood-forming stem cells and modifies them using a special tool called a lentiviral vector. This vector carries a healthy version of the ITGB2 gene into the cells. After modification, the stem cells are infused back into the patient with the goal of helping the immune system work better by correcting the genetic problem that causes the disease. The treatment is administered through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein.
Summary
There is currently one active clinical trial for Leukocyte Adhesion Deficiency-I, located in Spain. This trial is specifically designed for patients who have already participated in an earlier phase of research and received the investigational gene therapy. The focus is on long-term monitoring of safety and effectiveness, which reflects the careful and extended approach needed for gene therapy treatments. The trial uses RP-L201, a gene therapy approach that modifies the patient's own stem cells to address the underlying genetic cause of the condition. This represents an important area of research for this rare immune disorder, as it aims to provide a potentially lasting solution rather than just managing symptoms.
