Ignite Creation Date:
2024-05-05 @ 11:50 AM
Last Modification Date:
2024-10-26 @ 9:14 AM
Study NCT ID:
NCT00145613
Status:
COMPLETED
Last Update Posted:
2009-02-13
First Post:
2005-09-01
Brief Title:
Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Haploidentical Stem Cell Transplantation Utilizing T-Cell Depletion as Therapy for Patients With Refractory Hematological Malignancies
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:
Relapsed disease is the most common cause of death in children with hematological malignancies Patients who fail high-intensity conventional chemotherapeutic regimens or relapse after stem cell transplantation have a poor prognosis Toxicity from multiple therapies and elevated leukemictumor burden usually make these patients ineligible for the aggressive chemotherapy regimens required for conventional stem cell transplantation Alternative options are needed One type of treatment being explored is called haploidentical transplant
Conventional blood or bone marrow stem cell transplant involves destroying the patients diseased marrow with radiation or chemotherapy Healthy marrow from a donor is then infused into the patient where it migrates to the bone marrow space to begin generating new blood cells The best type of donor is a sibling or unrelated donor with an identical immune system HLA match However most patients do not have a matched sibling available andor are unable to identify an acceptable unrelated donor through the registries in a timely manner In addition the aggressive treatment required to prepare the body for these types of transplants can be too toxic for these highly pretreated patients Therefore doctors are investigating haploidentical transplant using stem cells from HLA partially matched family member donors
Although haploidentical transplant has proven curative in many patients this procedure has been hindered by significant complications primarily regimen-related toxicity including graft versus host disease GVHD and infection due to delayed immune reconstitution These can in part be due to certain white blood cells in the graft called T cells GVHD happens when the donor T cells recognize the patients the host body tissues are different and attack these cells Although too many T cells increase the possibility of GVHD too few may cause the recipients immune system to reconstitute slowly or the graft to fail to grow leaving the patient at high-risk for infection However the presence of T cells in the graft may offer a positive effect called graft versus malignancy or GVM With GVM the donor T cells recognize the patients malignant cells as diseased and in turn attack these diseased cells
For these reasons a primary focus for researchers is to engineer the graft to provide a T cell depleted product to reduce the risk of GVHD yet provide a sufficient number of cells to facilitate immune reconstitution graft integrity and GVM
In this study patients were given a haploidentical graft engineered to with specific T cell parameter values using the CliniMACS system A reduced intensity preparative regimen was used to reduce regimen-related toxicity and mortality The primary goal of this study is to evaluate overall survival in those who receive this study treatment
Conventional blood or bone marrow stem cell transplant involves destroying the patients diseased marrow with radiation or chemotherapy Healthy marrow from a donor is then infused into the patient where it migrates to the bone marrow space to begin generating new blood cells The best type of donor is a sibling or unrelated donor with an identical immune system HLA match However most patients do not have a matched sibling available andor are unable to identify an acceptable unrelated donor through the registries in a timely manner In addition the aggressive treatment required to prepare the body for these types of transplants can be too toxic for these highly pretreated patients Therefore doctors are investigating haploidentical transplant using stem cells from HLA partially matched family member donors
Although haploidentical transplant has proven curative in many patients this procedure has been hindered by significant complications primarily regimen-related toxicity including graft versus host disease GVHD and infection due to delayed immune reconstitution These can in part be due to certain white blood cells in the graft called T cells GVHD happens when the donor T cells recognize the patients the host body tissues are different and attack these cells Although too many T cells increase the possibility of GVHD too few may cause the recipients immune system to reconstitute slowly or the graft to fail to grow leaving the patient at high-risk for infection However the presence of T cells in the graft may offer a positive effect called graft versus malignancy or GVM With GVM the donor T cells recognize the patients malignant cells as diseased and in turn attack these diseased cells
For these reasons a primary focus for researchers is to engineer the graft to provide a T cell depleted product to reduce the risk of GVHD yet provide a sufficient number of cells to facilitate immune reconstitution graft integrity and GVM
In this study patients were given a haploidentical graft engineered to with specific T cell parameter values using the CliniMACS system A reduced intensity preparative regimen was used to reduce regimen-related toxicity and mortality The primary goal of this study is to evaluate overall survival in those who receive this study treatment
Detailed Description:
Secondary objectives for this protocol are to 1 assess the kinetics of lymphohematopoietic reconstitution and 2 describe the short and long-term up to 5 years post- transplant toxicity of haploidentical stem cell transplantation including GVHD in children with refractory hematological malignancies
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