Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Status:
Completed
Trial end date:
2009-02-01
Target enrollment:
Participant gender:
Summary
Wiskott - Aldrich syndrome (WAS) is a rare disorder curable only through allogeneic
hematopoietic stem cell transplantation. A mismatched family member is an option when no
human leukocyte antigen (HLA-immune system type) matched related or matched unrelated donor
is available.
This study will evaluate a novel therapeutic strategy for patients with WAS who undergo
haploidentical transplantation using a parental donor. To reduce the risk of
transplant-related toxicities, participants will receive a reduced intensity chemotherapy and
antibody regimen (conditioning treatment). Participants will then receive an infusion of
donor stem cells depleted of certain white blood cells called T- and B-lymphocytes. The stem
cell depletion processing will be done through the use of the investigational CliniMACS
device. A certain number of T-lymphocytes will be added back to the processed stem cell graft
prior to infusion into the recipient.
The primary objective of this study is to determine the safety of haploidentical
transplantation in WAS patients using this specified conditioning regimen and engineered
graft. Safety will be defined in terms of engraftment (meaning how well the graft grows and
functions after infusion) and regimen-related toxicity within the first 100 days after
transplant.