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A Single Arm, Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome (WAS)
Conditions
Interventions
OTL-103
Locations
2
United States
Children's Healthcare of Atlanta, Inc
Atlanta, Georgia, United States
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)
Milan, Italy
Start Date
January 21, 2019
Primary Completion Date
March 18, 2024
Completion Date
September 1, 2027
Last Updated
September 29, 2025
NCT01852370
NCT00909363
NCT03399461
NCT01347346
NCT01289847
NCT00885833
Lead Sponsor
Fondazione Telethon
Collaborators
Data Source & Attribution
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