Immunodeficiency – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:28:03 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Immunodeficiency – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Use of OTL-103 Gene Therapy and Drug Combination for Patients with Wiskott-Aldrich Syndrome https://clinicaltrials.eu/trial/study-on-the-use-of-otl-103-gene-therapy-and-drug-combination-for-patients-with-wiskott-aldrich-syndrome/ Wed, 29 Apr 2026 14:22:38 +0000 https://clinicaltrials.eu/trial/study-on-the-use-of-otl-103-gene-therapy-and-drug-combination-for-patients-with-wiskott-aldrich-syndrome/ This clinical trial is focused on studying a rare genetic disorder known as Wiskott-Aldrich Syndrome (WAS). This condition affects the immune system and can lead to problems such as bleeding and infections. The study is testing a new treatment called OTL-103, which is a type of gene therapy. Gene therapy involves using a patient’s own cells that are modified to correct the genetic defect causing the disease. In this case, the cells are treated with a special virus that carries the correct version of the gene responsible for WAS. The modified cells are then returned to the patient to help improve their immune system function.

The purpose of the study is to evaluate how effective and safe this new treatment is for people with WAS. Participants will receive the gene therapy and will be monitored for changes in their health, particularly looking at the number of bleeding events and infections they experience. The study will also involve other medications, including Busulfan, Fludarabine Phosphate, Plerixafor, Rituximab, and Lenograstim, which are used to prepare the body for the gene therapy and support the treatment process.

Throughout the study, participants will receive regular check-ups to assess their overall health and the effectiveness of the treatment. The study will last for several years, with key assessments at 12, 24, and 36 months after receiving the gene therapy. The goal is to see if the treatment can reduce the frequency of severe infections and bleeding episodes, as well as to ensure that it is safe for long-term use. Participants will be closely monitored for any side effects or complications related to the treatment.

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