Ignite Creation Date:
2024-05-05 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00029393
Status:
COMPLETED
Last Update Posted:
2016-07-29
First Post:
2002-01-10
Brief Title:
Induction of Stable Chimerism for Sickle Cell Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2008-01
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:
To investigate a modified hematopoeitic cell transplantation HCT procedure for sickle cell disease that significantly reduces the toxicity of HCT yet retains its therapeutic benefit
Detailed Description:
BACKGROUND
Hematopoietic cell transplantation HCT has curative potential for individuals with sickle cell disease While the results of conventional HCT have been good this treatment carries risks of significant short- term and longterm toxicities For this reason HCT has been reserved for children who have experienced severe symptoms that predict a poor outcome Of interest some patients developed stable donor-host hematopoietic chimerism after conventional HCT Due to a natural enrichment of donor erythrocytes in the blood those who developed stable chimerism had a significant clinical benefit even when there was a minority of donor cells These observations have paralleled efforts to develop less-toxic non-myeloablative preparative regiments for transplantation proved first in a canine model of transplantation and subsequently translated successfully in a clinical trial for older adults with hematological malignancies
DESIGN NARRATIVE
Multicenter open-label phase I-II study in 30 children with sickle cell disease that combines a non-myeloablative pre-transplant hematopoietic cell transplantation HCT therapy with modulated post-grafting immunosuppression to control host-versus-graft and graft-versus-host reactions The approach relies on the ability to establish and maintain donor-host chimerism The primary study endpoint is stable donor cell engraftment secondary endpoints measure the impact of therapy on sickle cell-related symptoms and end-organ damage disease-free survival patient survival graft-versus-host disease complications etc The trial will be conducted within the existing network of Comprehensive Sickle Cell Centers and will be centrally coordinated by the Sickle Cell Coordinating Center
Hematopoietic cell transplantation HCT has curative potential for individuals with sickle cell disease While the results of conventional HCT have been good this treatment carries risks of significant short- term and longterm toxicities For this reason HCT has been reserved for children who have experienced severe symptoms that predict a poor outcome Of interest some patients developed stable donor-host hematopoietic chimerism after conventional HCT Due to a natural enrichment of donor erythrocytes in the blood those who developed stable chimerism had a significant clinical benefit even when there was a minority of donor cells These observations have paralleled efforts to develop less-toxic non-myeloablative preparative regiments for transplantation proved first in a canine model of transplantation and subsequently translated successfully in a clinical trial for older adults with hematological malignancies
DESIGN NARRATIVE
Multicenter open-label phase I-II study in 30 children with sickle cell disease that combines a non-myeloablative pre-transplant hematopoietic cell transplantation HCT therapy with modulated post-grafting immunosuppression to control host-versus-graft and graft-versus-host reactions The approach relies on the ability to establish and maintain donor-host chimerism The primary study endpoint is stable donor cell engraftment secondary endpoints measure the impact of therapy on sickle cell-related symptoms and end-organ damage disease-free survival patient survival graft-versus-host disease complications etc The trial will be conducted within the existing network of Comprehensive Sickle Cell Centers and will be centrally coordinated by the Sickle Cell Coordinating Center
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U01HL068091 | NIH | None | httpsreporternihgovquickSearchU01HL068091 |