Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
|ClinicalTrials.gov Identifier: NCT01193283|
Recruitment Status : Completed
First Posted : September 1, 2010
Results First Posted : November 11, 2015
Last Update Posted : May 22, 2017
- Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard treatment for SAA involves injections of antithymocyte globulin (ATG) plus cyclosporine (CsA). This regimen has been shown to improve the blood counts in about two-thirds of patients. However, the ATG/CsA regimen has the following limitations: (a) the disease can come back (relapse) in about one-third of patients who improve initially; and (b) in about 10% to 15% of cases, certain types of bone marrow cancer (such as myelodysplasia and leukemia) can develop (called evolution). Experience with other drugs in SAA such as cyclophosphamide suggests that similar response rates to ATG/CsA can be achieved with a lower risk of relapse and clonal evolution. However, cyclophosphamide was found to have significant side effects in SAA when investigated over 10 years ago due to increase risk of fungal infections.
- Better antibiotic drugs against fungus have been developed and are widely used to treat patients who have low white blood cell counts and are at risk of developing infections. In SAA patients in particular, these newer antibiotics have had a large impact in preventing and treating fungus infections. Researchers are revisiting the use of cyclophosphamide in SAA treatment, and plan to give a lower dose of CsA in combination with the immune-suppressing drug cyclophosphamide, as well as antibiotics to protect against infections, as a possible treatment for the disease.
- To determine the safety and effectiveness of the combination of cyclophosphamide and cyclosporine in treating severe aplastic anemia that has not been treated with immunosuppressive therapy.
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia Neutropenia Pancytopenia Severe Aplastic Anemia||Drug: Cyclophosphamide Drug: Cyclosporine||Phase 1 Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia|
|Study Start Date :||August 2010|
|Primary Completion Date :||September 2014|
|Study Completion Date :||September 2014|
U.S. FDA Resources
Experimental: SAA hematologic response
Treatment-naive severe aplastic anemia patients will receive a low dose of cyclophosphamide (120mg/kg) and low dose cyclosporine ( target therapeutic level of 100-200 micrograms per liter). Cyclophosphamide will be given once daily for 4 doses. Cyclosporine will be started after cyclophosphamide completion, cyclosporine will be given twice daily. The dosing will be modified to attain the therapeutic level.
30 my/kg for 4 days
Other Name: CytoxanDrug: Cyclosporine
daily to a trough of 100 t0 200 ng/ml
- Blood Counts and Adverse Event Profile After 6 Months of Treatment. [ Time Frame: 6 months ]The safety endpoint will be toxicity profile after 6 months of treatment. The efficacy endpoint is complete response rate at 6 months, with complete response defined as blood counts no longer meeting the standard criteria for severe pancytopenia in severe aplastic anemia.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01193283
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Danielle M Townsley, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|