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: Gene Therapy Follow-Up
We are monitoring the long-term safety of a gene therapy in patients with Wiskott-Aldrich Syndrome who received a specific stem cell treatment. This study helps us understand how the therapy affects their health over time.
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..
- Are you willing to return for the follow-up study period?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Autologous CD34+ Cells Transduced with the W1.6_hWASP_WPRE (VSVg) Lentiviral Vector
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.
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Hopital Necker Enfants Malades
Département Biothérapies
Montrouge, France
Sponsor: Genethon
Last updated: Nov 27, 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.