This clinical trial focuses on a rare genetic disorder called Wiskott-Aldrich Syndrome (WAS). This condition affects the immune system and can lead to issues such as bleeding, infections, and eczema. The study is examining a treatment known as Telethon003, which is a type of gene therapy. Gene therapy involves using a patient’s own cells, which are modified outside the body to correct the genetic defect, and then infused back into the patient. The specific treatment being studied is called etuvetidigene autotemcel, also known as OTL-103.

The purpose of this study is to understand the long-term safety and effectiveness of the Telethon003 treatment for individuals who have already received it. Participants in the study will be monitored over a long period to see how well they are doing after the treatment. This includes checking for any side effects or health issues that might arise, as well as assessing their overall health and quality of life. The study will also look at how the treatment affects survival rates and the frequency of infections and bleeding episodes.

Throughout the study, various health aspects will be evaluated, such as the occurrence of eczema, autoimmune conditions, and blood disorders. The study will also track changes in growth for children, and assess quality of life using specific questionnaires. The goal is to gather comprehensive information on how the treatment impacts the health and well-being of those with Wiskott-Aldrich Syndrome over time.