Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
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.
|Wiskott-Aldrich Syndrome||Procedure: Hematopoietic stem cell transplantation Device: Miltenyi CliniMACS selection system Drug: Fludarabine, Melphalan, Thiotepa||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study|
- To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning [ Time Frame: March 2010 ]
|Study Start Date:||May 2005|
|Study Completion Date:||February 2009|
|Primary Completion Date:||July 2008 (Final data collection date for primary outcome measure)|
Procedure: Hematopoietic stem cell transplantation
To determine the safety in regards to engraftment and toxicity within 100 days of infusing a haploidentical T- and B-cell depleted hematopoietic stem cell graft into patients with Wiskott-Aldrich syndrome who have received a reduced intensity conditioning regimen.
Other Names:Device: Miltenyi CliniMACS selection system
This system depletes the hematopoietic stem cell graft of T and B lymphocytes.Drug: Fludarabine, Melphalan, Thiotepa
Participants will receive a reduced intensity conditioning regimen consisting of Fludarabine, Melphalan, Thiotepa, and OKT3 prior to receipt of the haploidentical stem cell graft. Rituximab will be given in an effort to prevent PTLPD. In addition to T-cell depletion of the donor product, cyclosporine will be given for GVHD prophylaxis.
Secondary Objectives in this trial include the following:
- To estimate the survival of study recipients at one year after infusion of the T- and B-lymphocyte depleted stem cell graft.
- To assess if the study treatment enables the recipient to generate normal donor-derived B-cell numbers and endogenous IgM, IgG, and IgA production, resulting in a reduction/elimination of the need for intravenous immunoglobulin infusions.
- To determine if the study treatment results in the ability of the research participant to generate normal donor-derived T cell response and natural killer (NK) cell numbers and function.
- To describe the incidence of Epstein-Barr virus-lymphoproliferative disease (EBV-LPD) in these transplant recipients.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00160355
|United States, Tennessee|
|St. Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38105|
|Principal Investigator:||Kimberly Kasow, DO||St. Jude Children's Research Hospital|