Wiskott-Aldrich Syndrome (WAS) Clinical Trial
Official title:
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
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.
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT00774358 -
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
|
Phase 1 |