Ignite Creation Date:
2024-05-05 @ 11:31 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00065260
Status:
COMPLETED
Last Update Posted:
2021-07-21
First Post:
2003-07-18
Brief Title:
Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATGCsA Treatment
Status:
COMPLETED
Status Verified Date:
2021-06
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:
Severe aplastic anemia characterized by pancytopenia and a hypocellular bone marrow is effectively treated by immunosuppressive therapy usually a combination of antithymocyte globulin ATG and cyclosporine CsA Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation However approximately 13 of patients will not show blood count improvement after ATGCsA General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression Furthermore analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG even when transfusion-independence is achieved have a markedly worse prognosis than patients with robust hematologic improvement The management of such cases is uncertain
This study will enroll patients who are either refractory to h-ATG continued severe pancytopenia or who have only modest improvement in blood counts weak hematologic responders to receive a further immunosuppressive therapy delivered either as rabbit ATG Thymoglobulin r-ATG or a humanized monoclonal antibody to T-cells alemtuzumab Campath-1H Primary endpoint will be response rate at 3 months defined as no longer meeting criteria for severe aplastic anemia Relapse robustness of hematopoietic recovery at 3 months survival and clonal evolution to paroxysmal nocturnal hemoglobinuria PNH myelodysplasia and acute leukemia will be the secondary endpoints
This study will enroll patients who are either refractory to h-ATG continued severe pancytopenia or who have only modest improvement in blood counts weak hematologic responders to receive a further immunosuppressive therapy delivered either as rabbit ATG Thymoglobulin r-ATG or a humanized monoclonal antibody to T-cells alemtuzumab Campath-1H Primary endpoint will be response rate at 3 months defined as no longer meeting criteria for severe aplastic anemia Relapse robustness of hematopoietic recovery at 3 months survival and clonal evolution to paroxysmal nocturnal hemoglobinuria PNH myelodysplasia and acute leukemia will be the secondary endpoints
Detailed Description:
Severe aplastic anemia characterized by pancytopenia and a hypocellular bone marrow is effectively treated by immunosuppressive therapy usually a combination of antithymocyte globulin ATG and cyclosporine CsA Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation However approximately 13 of patients will not show blood count improvement after ATGCsA General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression Furthermore analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG even when transfusion-independence is achieved have a markedly worse prognosis than patients with robust hematologic improvement The management of such cases is uncertain
This study will enroll patients who are either refractory to h-ATG continued severe pancytopenia or who have only modest improvement in blood counts weak hematologic responders to receive further immunosuppressive therapy delivered either as rabbit ATG Thymoglobulin r-ATG or a humanized monoclonal antibody to T-cells alemtuzumab Campath-1H Primary endpoint will be response rate at 6 months defined as no longer meeting criteria for severe aplastic anemia Relapse robustness of hematopoietic recovery at 6 months survival and clonal evolution to paroxysmal nocturnal hemoglobinuria PNH myelodysplasia and acute leukemia will be the secondary endpoints
This study will enroll patients who are either refractory to h-ATG continued severe pancytopenia or who have only modest improvement in blood counts weak hematologic responders to receive further immunosuppressive therapy delivered either as rabbit ATG Thymoglobulin r-ATG or a humanized monoclonal antibody to T-cells alemtuzumab Campath-1H Primary endpoint will be response rate at 6 months defined as no longer meeting criteria for severe aplastic anemia Relapse robustness of hematopoietic recovery at 6 months survival and clonal evolution to paroxysmal nocturnal hemoglobinuria PNH myelodysplasia and acute leukemia will be the secondary endpoints
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 |
|---|---|---|---|
| 03-H-0249 | None | None | None |