Ignite Creation Date:
2024-05-05 @ 11:53 AM
Last Modification Date:
2024-10-26 @ 9:15 AM
Study NCT ID:
NCT00160355
Status:
COMPLETED
Last Update Posted:
2017-04-26
First Post:
2005-09-08
Brief Title:
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome A Pilot Study
Status:
COMPLETED
Status Verified Date:
2009-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
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
Detailed Description:
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 reductionelimination 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
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 reductionelimination 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None