Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00034528
Status:
TERMINATED
Last Update Posted:
2013-05-01
First Post:
2002-04-30
Brief Title:
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institute of Allergy and Infectious Diseases NIAID
Study Overview
Official Title:
Allogeneic Stem Cell Transplantation Following Nonmyeloablative Chemotherapy in Patients With Hemoglobinopathies
Status:
TERMINATED
Status Verified Date:
2013-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to slow recruitment
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation
Detailed Description:
Hemoglobinopathies such as sickle cell disease and thalassemia major are genetic diseases associated with significant morbidity and premature death Allogeneic bone marrow transplantation BMT is the only potential cure for severe hemoglobinopathies Typical regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient hematopoiesis and to prevent graft rejection The widespread use of this treatment has been limited by toxicity risk of end-organ damage and donor availability This study will use a nonmyeloablative regimen of fludarabine and busulfan to attempt to generate consistent engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy
G-CSF mobilization of the donors peripheral blood white blood cells will precede donor apheresis A nonmyeloablative conditioning regimen of fludarabine and busulfan will be administered to patients prior to allogeneic peripheral blood stem cell infusions FK506 and prednisone will be administered for graft versus host disease GVHD prophylaxis Patients will be evaluated for engraftment donor host hematopoietic chimerism toxicity and hemoglobinopathy
G-CSF mobilization of the donors peripheral blood white blood cells will precede donor apheresis A nonmyeloablative conditioning regimen of fludarabine and busulfan will be administered to patients prior to allogeneic peripheral blood stem cell infusions FK506 and prednisone will be administered for graft versus host disease GVHD prophylaxis Patients will be evaluated for engraftment donor host hematopoietic chimerism toxicity and hemoglobinopathy
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 |
|---|---|---|---|
| P01 A 129530 | None | None | None |