Ignite Creation Date:
2024-05-05 @ 12:07 PM
Last Modification Date:
2024-10-26 @ 9:20 AM
Study NCT ID:
NCT00244010
Status:
COMPLETED
Last Update Posted:
2017-05-30
First Post:
2005-10-24
Brief Title:
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Sponsor:
St Jude Childrens Research Hospital
Organization:
St Jude Childrens Research Hospital
Study Overview
Official Title:
Hematopoietic Stem Cell Transplantation HSCT From Partially Matched Family Donors for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias 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:
Due to an overall and disease free survival of 85 to 100 allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia SAA Unfortunately only about 25 of patients have such a donor For patients with SAA lacking a matched sibling donor immunosuppressive therapy is the current treatment of choice Approximately 70 of these patients have a complete or partial response to immunosuppressive therapy achieving transfusion independence andor growth factor independence
For the approximately 30 of patients who do not respond to immunosuppressive therapy or experience recurrence alternative donor matched unrelated partially matched family member transplantation is a treatment option However graft rejection and graft-versus-host-disease GVHD are significant barriers to success decreasing event-free survival to 30 to 50
This study offers stem cell transplantation using a partially matched family member haploidentical donor to those patients with no available HLA-matched sibling or matched unrelated donor In an attempt to reduce GVHD and regimen-related toxicity while maintaining adequate engraftment we plan to infuse a highly purified stem cell graft The Miltenyi Biotec CliniMACS CD3 depletion system will be used to derive a defined allogeneic graft highly enriched for CD34 hematopoietic cells and depleted of CD3 T-lymphocytes from G-CSF mobilized donor-derived peripheral blood stem cells
Patients 21 years of age and younger with refractory cytopenias are also eligible for this protocol as there are no other potentially curative therapies currently available for these conditions
The primary objective of this study is to evaluate the safety of transplantation using a haploidentical donor product engineered to targeted cell counts using the investigational CliniMACS device for patients with refractory severe aplastic anemia SAA or refractory cytopenias The treatment plan would be considered unsafe if we can find this type of procedure is associated with a significantly higher treatment failure rate Treatment failure is defined as any occurrence of the following events overall grade III-IV acute GVHD graft failure or death due to any cause within 100 days after transplant
For the approximately 30 of patients who do not respond to immunosuppressive therapy or experience recurrence alternative donor matched unrelated partially matched family member transplantation is a treatment option However graft rejection and graft-versus-host-disease GVHD are significant barriers to success decreasing event-free survival to 30 to 50
This study offers stem cell transplantation using a partially matched family member haploidentical donor to those patients with no available HLA-matched sibling or matched unrelated donor In an attempt to reduce GVHD and regimen-related toxicity while maintaining adequate engraftment we plan to infuse a highly purified stem cell graft The Miltenyi Biotec CliniMACS CD3 depletion system will be used to derive a defined allogeneic graft highly enriched for CD34 hematopoietic cells and depleted of CD3 T-lymphocytes from G-CSF mobilized donor-derived peripheral blood stem cells
Patients 21 years of age and younger with refractory cytopenias are also eligible for this protocol as there are no other potentially curative therapies currently available for these conditions
The primary objective of this study is to evaluate the safety of transplantation using a haploidentical donor product engineered to targeted cell counts using the investigational CliniMACS device for patients with refractory severe aplastic anemia SAA or refractory cytopenias The treatment plan would be considered unsafe if we can find this type of procedure is associated with a significantly higher treatment failure rate Treatment failure is defined as any occurrence of the following events overall grade III-IV acute GVHD graft failure or death due to any cause within 100 days after transplant
Detailed Description:
Secondary objectives for this protocol include the following
To observe the degree of hematopoietic chimerism in T-cells during the first year posttransplant
To observe the relative proportions of donorhost T-regulatory cells during the first year posttransplant
To monitor rates of acute and chronic GVHD during the first year posttransplant
To observe the degree of hematopoietic chimerism in T-cells during the first year posttransplant
To observe the relative proportions of donorhost T-regulatory cells during the first year posttransplant
To monitor rates of acute and chronic GVHD during the first year posttransplant
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| Severe Aplastic Anemia | None | None | None |
| Cytopenias | None | None | None |