Ignite Creation Date:
2024-05-05 @ 10:17 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001749
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Medical Treatment for Diamond Blackfan Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A
Status:
COMPLETED
Status Verified Date:
2005-07
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:
Diamond Blackfan anemia DBA is a condition in which the bone marrow is underdeveloped DBA is considered a congenital disease meaning patients are born with it In DBA there is a lack of cells that give rise to red blood cells The other elements produced in the bone marrow such as white blood cells and platelets are normal
Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure relapse side-effects increased morbidity and even death Two drugs antithymocyte globulin ATG and cyclosporin have been used to treat DBA but have only provided occasional responses No study has ever combined these two drugs for the treatment of DBA
This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA
Standard treatments used for this disorder such as steroids and bone marrow transplants are associated with failure relapse side-effects increased morbidity and even death Two drugs antithymocyte globulin ATG and cyclosporin have been used to treat DBA but have only provided occasional responses No study has ever combined these two drugs for the treatment of DBA
This study is designed to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of DBA
Detailed Description:
Diamond Blackfan anemia DBA is a constitutional pure red cell aplasia of unknown etiology There is laboratory evidence for an immune mechanism and most patients respond to corticosteroids However the relapse and failure rate are high and corticosteroids are associated with many short and long term side effects Patients who do not respond or who do not tolerate corticosteriods require lifelong red blood cell transfusion and iron chelation therapy Allogeneic bone marrow transplantation is an option for those with a related histocompatible donor but this procedure is associated with high mortality and morbidity Other therapies have been tried without general success Occasional responses to either ATG or cyclosporine have been reported but no study has used both ATG and cyclosporine In other bloodbone marrow disorders of immune etiology these drugs have synergistic effects We propose a Phase II study to explore the combined use of ATG and cyclosporine as a rational approach to the treatment of Diamond Blackfan anemia
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 |
|---|---|---|---|
| 98-H-0144 | None | None | None |