NCT01515462

Gene Therapy for Wiskott-Aldrich Syndrome

A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Fondazione Telethon · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.

Detailed description

Wiskott-Aldrich Syndrome (WAS) is an X-linked primary immunodeficiency caused by mutations in the WAS gene which encodes the WAS protein (WASP), a cytoskeletal regulator which is expressed exclusively in hematopoietic cells.

Conditions

• Wiskott-Aldrich Syndrome (WAS)

Interventions

Type	Name	Description
GENETIC	TLT003	TLT003 is an autologous CD34+ cells collected from bone marrow and/or peripheral blood and transduced with a lentiviral vector encoding Wiskott-Aldrich syndrome (WAS) protein

Timeline

• Start date: 2010-04-20
• Primary completion: 2023-10-04
• Completion: 2023-10-04
• First posted: 2012-01-24
• Last updated: 2025-04-04
• Results posted: 2025-04-04

Locations

1 site across 1 country: Italy