Ignite Creation Date:
2024-05-05 @ 10:17 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001964
Status:
COMPLETED
Last Update Posted:
2021-03-16
First Post:
2000-01-18
Brief Title:
Combination Therapy of Severe Aplastic Anemia
Sponsor:
National Heart Lung and Blood Institute NHLBI
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Treatment of Severe Aplastic Anemia With Combined Immunosuppression Antithymocyte Globulin ATG and Cyclosporine A CSA and Mycophenolate Mofetil MMF
Status:
COMPLETED
Status Verified Date:
2021-03
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 test the safety and effectiveness of a combination of three drugs in treating severe aplastic anemia and preventing its recurrence Two drugs used in this trial ATG and cyclosporine are standard combination therapy for aplastic anemia This study will try to improve this therapy in three ways 1 by altering the drug regimen to allow the drugs to work better 2 by reducing the risk of kidney damage and 3 by adding a third drug mycophenolate mofetil to try to prevent disease relapse
Patients with severe aplastic anemia who do not have a suitable bone marrow donor or who decline bone marrow transplantation may participate in this study Patients will have a skin test for ATG allergy chest X-ray blood test and bone marrow aspiration before treatment begins ATG will then be started infused through a vein continuously for 4 days Ten days after ATG is stopped cyclosporine treatment will begin taken twice a day by mouth in either liquid or capsule form and will continue for 6 months Also in the first 2 weeks of treatment patients will be given a full dose of corticosteroid prednisone to prevent serum sickness that could develop as a side effect of ATG therapy The dosage will be decreased after that Mycophenolate will be started at the same time as ATG in two daily doses by mouth and will continue for 18 months
Patients will be hospitalized at the beginning of the study During this time blood will be drawn at 3-week intervals and a bone marrow examination will be repeated 3 months after treatment has begun Additional tests including X-rays may be required After hospital discharge patients will be followed on an outpatient basis at 3-month intervals The patients own physician will perform blood tests weekly and kidney and liver function tests every 2 weeks during cyclosporine therapy Transfusions may be required initially
Patients with severe aplastic anemia who do not have a suitable bone marrow donor or who decline bone marrow transplantation may participate in this study Patients will have a skin test for ATG allergy chest X-ray blood test and bone marrow aspiration before treatment begins ATG will then be started infused through a vein continuously for 4 days Ten days after ATG is stopped cyclosporine treatment will begin taken twice a day by mouth in either liquid or capsule form and will continue for 6 months Also in the first 2 weeks of treatment patients will be given a full dose of corticosteroid prednisone to prevent serum sickness that could develop as a side effect of ATG therapy The dosage will be decreased after that Mycophenolate will be started at the same time as ATG in two daily doses by mouth and will continue for 18 months
Patients will be hospitalized at the beginning of the study During this time blood will be drawn at 3-week intervals and a bone marrow examination will be repeated 3 months after treatment has begun Additional tests including X-rays may be required After hospital discharge patients will be followed on an outpatient basis at 3-month intervals The patients own physician will perform blood tests weekly and kidney and liver function tests every 2 weeks during cyclosporine therapy Transfusions may be required initially
Detailed Description:
Severe acquired aplastic anemia SAA has a poor prognosis if untreated Bone marrow transplantation is available to only a minority of patients due to lack of a matched sibling donor advanced age of the patient or cost Clinical studies at NIH and elsewhere have demonstrated excellent response rates and improved survival with immunosuppressive treatments Laboratory data implicate underlying cytotoxic T-lymphocyte-mediated suppression of hematopoiesis as the likely proximal cause of disease in most patients In earlier clinical protocols we treated SAA with cyclosporine A CSA 86-H-0007 antithymocyte globulin ATG 87-H-0124 and combined ATG and CSA 90-H-0146 While intensive immunosuppression is most effective relapse is common and some patients also develop second hematologic complications like myelodysplasia In this protocol we modify our regimen by delaying the introduction of cyclosporine to promote ATG tolerizing effects and adding mycophenolate mofetil MMF a new agent that like ATG may be relatively specific for activated lymphocytes in an effort to reduce the high relapse rate
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
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 |
|---|---|---|---|
| 00-H-0032 | None | None | None |