Ignite Creation Date:
2024-05-05 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00029380
Status:
COMPLETED
Last Update Posted:
2016-07-29
First Post:
2002-01-10
Brief Title:
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Heart Lung and Blood Institute NHLBI
Study Overview
Official Title:
Sibling Donor Cord Blood Banking and Transplantation
Status:
COMPLETED
Status Verified Date:
2008-09
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:
This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia
Detailed Description:
BACKGROUND
During the past decade a number of advances have been made in the treatment of patients with sickle cell anemia and thalassemia Among these advances is allogeneic bone marrow transplantation which is the only current treatment that offers a potential for cure In sickle cell anemia transplantation has been performed in patients who have had advanced organ damage In thalassemia transplantation has been performed before having any evidence of iron-related tissue damage Due to concerns over engraftment and graft versus host disease GVHD transplants for patients with hemoglobinopathies have been limited to situations in which a human leukocyte antigen HLA compatible donor existed Unfortunately an HLA-matched related donor is often not available Umbilical cord blood UCB a recently recognized source of hematopoietic stem cells has been used to successfully transplant bone marrow to over 500 patients The potential advantage of cord blood over other donor sources of stem cells is the minimal risk of high-grade GVHD even without complete HLA compatibility
DESIGN NARRATIVE
This study will establish a national sibling donor cord blood SDCB program evaluate its use in a multi-center pilot study of transplantation and develop a Web-based data management system to support these two projects A multi-center pilot study was conducted on cord blood transplantation in children with either sickle cell disease or thalassemia The investigators tested the hypothesis that a novel immunosuppressive conditioning regimen fludarabine cyclophosphamide and busulfan and post transplant therapy mycophenolate mofetil and cyclosporine would improve engraftment rates and prevent disease recurrence The effect of SDCB transplantation on hematologic parameters and GVHD was monitored Enrollment in the study was suspended on December 29 2003 The protocol was revised replacing the previous conditioning regimen of fludarabine busulfan and cyclophosphamide with a more conventional regimen of rabbit anti-thymocyte globulin Sangstat busulfan and cyclophosphamide The revised protocol is open for enrollment
During the past decade a number of advances have been made in the treatment of patients with sickle cell anemia and thalassemia Among these advances is allogeneic bone marrow transplantation which is the only current treatment that offers a potential for cure In sickle cell anemia transplantation has been performed in patients who have had advanced organ damage In thalassemia transplantation has been performed before having any evidence of iron-related tissue damage Due to concerns over engraftment and graft versus host disease GVHD transplants for patients with hemoglobinopathies have been limited to situations in which a human leukocyte antigen HLA compatible donor existed Unfortunately an HLA-matched related donor is often not available Umbilical cord blood UCB a recently recognized source of hematopoietic stem cells has been used to successfully transplant bone marrow to over 500 patients The potential advantage of cord blood over other donor sources of stem cells is the minimal risk of high-grade GVHD even without complete HLA compatibility
DESIGN NARRATIVE
This study will establish a national sibling donor cord blood SDCB program evaluate its use in a multi-center pilot study of transplantation and develop a Web-based data management system to support these two projects A multi-center pilot study was conducted on cord blood transplantation in children with either sickle cell disease or thalassemia The investigators tested the hypothesis that a novel immunosuppressive conditioning regimen fludarabine cyclophosphamide and busulfan and post transplant therapy mycophenolate mofetil and cyclosporine would improve engraftment rates and prevent disease recurrence The effect of SDCB transplantation on hematologic parameters and GVHD was monitored Enrollment in the study was suspended on December 29 2003 The protocol was revised replacing the previous conditioning regimen of fludarabine busulfan and cyclophosphamide with a more conventional regimen of rabbit anti-thymocyte globulin Sangstat busulfan and cyclophosphamide The revised protocol is open for enrollment
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 |
|---|---|---|---|
| U01HL061877 | NIH | None | httpsreporternihgovquickSearchU01HL061877 |