Wiskott-Aldrich Syndrome
Wiskott-Aldrich Syndrome (WAS) is a rare genetic disorder characterized by eczema, recurrent infections due to immune deficiency, and a tendency to bleed easily due to low platelet counts.
Wiskott-Aldrich Syndrome: Long-term Study of Gene Therapy
We are examining the long-term safety and effectiveness of a gene therapy treatment for individuals with Wiskott-Aldrich Syndrome. This study aims to provide insights into how well the treatment works over time.
>2 yearsConfirmation phase (III)No PlaceboPartially RemoteInvestigational MedicinesInjectionHematologyInfectious Diseases
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Have you been treated with Telethon003?
- Are you able to provide informed consent for yourself or have a parent/guardian who can?
- Are you willing to attend all study visits as scheduled?
- Are you currently participating in another clinical study with an investigational drug?
- Have you taken any other investigational drug or therapy like a stem cell transplant recently?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Otl-103
Etuvetidigene Autotemcel
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
Loading map...
San Raffaele Hospital
Pediatric Immunohematology Unit
Vimodrone, Italy
Sponsor: Fondazione Telethon Ets
Last updated: Nov 26, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.