Wiskott-Aldrich syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 11:15:38 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Wiskott-Aldrich syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Long-term Safety and Efficacy Study of Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-etuvetidigene-autotemcel-for-patients-with-wiskott-aldrich-syndrome/ Wed, 29 Apr 2026 15:04:26 +0000 https://clinicaltrials.eu/trial/long-term-safety-and-efficacy-study-of-etuvetidigene-autotemcel-for-patients-with-wiskott-aldrich-syndrome/ 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.

]]> Long-Term Safety Study of Gene Therapy with Etuvetidigene Autotemcel for Patients with Wiskott-Aldrich Syndrome https://clinicaltrials.eu/trial/long-term-safety-study-of-gene-therapy-with-etuvetidigene-autotemcel-for-patients-with-wiskott-aldrich-syndrome/ Wed, 29 Apr 2026 15:01:26 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-gene-therapy-with-etuvetidigene-autotemcel-for-patients-with-wiskott-aldrich-syndrome/ This clinical trial focuses on the long-term safety of a gene therapy treatment for patients with Wiskott-Aldrich Syndrome, a rare genetic disorder that affects the immune system and blood clotting. The treatment involves using the patient’s own blood stem cells, which are modified outside the body with a special virus to carry a healthy version of the WASP gene. This modified cell product is known as etuvetidigene autotemcel and is given to patients through an infusion.

The purpose of the study is to monitor patients who have received this gene therapy to ensure its safety over a long period. Participants will have regular check-ups to assess their overall health, including their immune system function and blood cell counts. The study will also look for any delayed side effects, such as new health issues that might develop years after the treatment.

Throughout the study, researchers will evaluate the need for additional treatments, such as antibiotics or blood transfusions, and will perform optional tests to check the health of the bone marrow. The study aims to provide valuable information on the long-term effects of this gene therapy for Wiskott-Aldrich Syndrome and help improve future treatments for this condition.

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