Note: This study has reached the target number of patients to be treated, and accrual for the trial is currently closed.
Wiskott-Aldrich syndrome (WAS) is a rare genetic immunodeficiency that keeps a child's
immune system from functioning properly. It also makes it difficult for a
child's bone marrow to produce platelets, making a child prone to bleeding. The
traditional standard therapy for Wiskott-Aldrich syndrome is allogeneic stem
cell transplant, but this often has suboptimal outcomes.
In our Wiskott-Aldrich syndrome gene therapy trial, autologous
CD34+ bone marrow or peripheral blood stem cells are collected from the patient
and transduced ex vivo with a lentiviral gene transfer vector, then infused
back into the patient after high dose chemotherapy with busulfan and
fludarabine. Such an approach is suitable for patients who lack well-matched
donors and avoids allogeneic complications such as graft-versus-host disease (GVHD)
This trial will test whether this approach is safe and
whether gene transfer will lead to immunologic and hematologic reconstitution.
The costs of research aspects of the protocol are provided for patients treated
on the trial by the Gene Therapy Resource Program, NHLBI and NIH.
For subjects < 5 years of age:
For subjects 5 years of age or
Subjects who have previously
undergone allogeneic transplant and demonstrate: