Clinical Trial

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This is an open-label, single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy. OTL-103 consists of autologous CD34+ hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector.
Protocol Amendment History 14 amendments
This ClinicalTrials.gov record has been amended 14 times since 2019-02-11; most recent amendment 2025-09-23.
Status change: Recruiting → Active, Not Recruiting 2022-09-27
Status change: Active, Not Recruiting → Recruiting 2021-09-09
Status change: Recruiting → Active, Not Recruiting 2020-04-08
Trial Details
NCT Number NCT03837483
Lead Sponsor Fondazione Telethon
Collaborators: Ospedale San Raffaele
Conditions Wiskott-Aldrich Syndrome
Enrollment 10 participants
Start Date 2019-01-21
Primary Completion 2024-03-18 (estimated)
Study Completion 2027-09 (estimated)
Updated on ClinicalTrials.gov 2025-09-29