OTL-103
Sponsors
Fondazione Telethon Ets, Fondazione Telethon
Conditions
Wiskott-Aldrich SyndromeWiskott-Aldrich syndrome (WAS)
Phase 3
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
Active, not recruitingNCT03837483
Start: 2019-01-21End: 2027-09-30Updated: 2025-09-29
A Single Arm, Open Label Clinical Study 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)
Not yet recruitingCTIS2024-517792-20-00
Target: 9Updated: 2025-09-23
A Long-term Follow-up Study for Subjects Previously Treated with Autologous ex vivo Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
RecruitingCTIS2024-518369-92-00
Start: 2025-06-25Target: 38Updated: 2025-06-19