Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001626
Status:
COMPLETED
Last Update Posted:
2020-09-22
First Post:
1999-11-03
Brief Title:
Comparing Therapies for the Treatment of 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 Antithymocyte Globulin and Cyclosporine Versus Cyclophosphamide and Cyclosporine in the Treatment of Severe Aplastic Anemia
Status:
COMPLETED
Status Verified Date:
2020-09
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:
Severe Aplastic Anemia SAA is a rare and very serious blood disorder in which the bone marrow stops producing the cells which make up blood red blood cells white blood cells and platelets
Researchers believe this is caused by an autoimmune reaction a condition in which the natural defense system of the body begins attacking itself In SAA the immune system begins attacking the bone marrow Red blood cells are responsible for carrying oxygen to all of the organ systems in the body and low numbers anemia can cause difficulty breathing and fatigue Platelets are responsible for normal blood clotting and low numbers can result in easy bruising and bleeding which can be deadly White blood cells are responsible for fighting infections and low numbers of these can lead to frequent infections the most common cause of death in patients with aplastic anemia
SAA can be treated by bone marrow transplant BMT or by drugs designed to slow down the immune system immunosuppressants BMT can be successful but it requires a donor with matched bone marrow making this therapy available only to a few patients BMT with unmatched bone marrow can fail and cause dangerous side effects
Presently the two drugs used to treat SAA by slowing down the immune system immunosuppression are antithymocyte globulin ATG and cyclosporin A CSA When used in combination these two drugs can improve most patients condition However one third of the patients who respond to this therapy experience a relapse of SAA In addition some patients treated with ATGCSA can later develop other disorders of the blood
Recently researchers have found that another immunosuppressive drug called cyclophosphamide has been successful at treating patients with SAA In addition patients treated with cyclophosphamide do not experience relapses or develop other disorders of the blood
In this study researchers would like to compare the combinations of antithymocyte globulin ATG and cyclosporin A CSA to cyclophosphamide and cyclosporin A CSA for the treatment of SAA
Researchers believe this is caused by an autoimmune reaction a condition in which the natural defense system of the body begins attacking itself In SAA the immune system begins attacking the bone marrow Red blood cells are responsible for carrying oxygen to all of the organ systems in the body and low numbers anemia can cause difficulty breathing and fatigue Platelets are responsible for normal blood clotting and low numbers can result in easy bruising and bleeding which can be deadly White blood cells are responsible for fighting infections and low numbers of these can lead to frequent infections the most common cause of death in patients with aplastic anemia
SAA can be treated by bone marrow transplant BMT or by drugs designed to slow down the immune system immunosuppressants BMT can be successful but it requires a donor with matched bone marrow making this therapy available only to a few patients BMT with unmatched bone marrow can fail and cause dangerous side effects
Presently the two drugs used to treat SAA by slowing down the immune system immunosuppression are antithymocyte globulin ATG and cyclosporin A CSA When used in combination these two drugs can improve most patients condition However one third of the patients who respond to this therapy experience a relapse of SAA In addition some patients treated with ATGCSA can later develop other disorders of the blood
Recently researchers have found that another immunosuppressive drug called cyclophosphamide has been successful at treating patients with SAA In addition patients treated with cyclophosphamide do not experience relapses or develop other disorders of the blood
In this study researchers would like to compare the combinations of antithymocyte globulin ATG and cyclosporin A CSA to cyclophosphamide and cyclosporin A CSA for the treatment of SAA
Detailed Description:
Severe aplastic anemia SAA is a disorder with a poor prognosis if untreated Current accepted therapeutic strategies include bone marrow transplantation BMT and immunosuppression both offering cure or amelioration in the majority of patients Although BMT is successful using human leukocyte antigen HLA matched sibling bone marrow the 25 probability of finding an HLA identical sibling within a family renders this approach available to only a minority of patients BMT utilizing HLA-matched unrelated donors carries a high risk of treatment failure along with considerable toxicity While combined immunosuppression with both antithymocyte globulin ATG and cyclosporine A CSA produces hematologic improvement in most patients relapse is common occurring in about a third of responders Late evolution of aplastic anemia to other serious hematologic disorders is a significant problem following successful treatment with ATGCSA with paroxysmal nocturnal hemoglobinuria PNH occurs in approximately 13 myelodysplasia in about 10 and acute leukemia in about 7 Recently results of immunosuppression in SAA with another potent immunosuppressive agent cyclophosphamide were reported in 10 patients In this small group the overall response rate was similar to that seen with ATGCSA but relapse and late clonal disease were not seen during a median follow-up of greater than 10 years In the larger randomized trial proposed here we will compare sustained hematologic response rates to either conventional immunosuppression with ATGCSA or high dose cyclophosphamide and CSA Secondary endpoints include response duration event free survival and overall survival
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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 |
|---|---|---|---|
| 97-H-0117 | None | None | None |