A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01187017|
Recruitment Status : Completed
First Posted : August 23, 2010
Results First Posted : March 2, 2016
Last Update Posted : March 2, 2016
- Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard initial treatment for SAA involves injections of antithymocyte globulin (ATG) plus cyclosporine (CsA). Patients with SAA who do not respond to initial treatment with ATG (refractory) have a high risk of dying without additional treatment. In these cases, for those who do not have a matched bone marrow transplant donor there is no well-defined standard therapy. In our experience with patients who do not respond to horse ATG + CsA, only about one-third of patients who are re-treated with rabbit ATG + CsA improve. Experience with cyclophosphamide in the treatment of refractory severe aplastic anemia suggests that this drug is able to improve blood counts in about 50% of cases. However, the cyclophosphamide regimen has been associated with a significant infection risk (mostly caused by fungus) in studies conducted over 10 years ago due to the lowering of the white blood cell levels.
- 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 at lower doses to minimize its side effects given in combination with another immune suppressant, fludarabine.
- To determine the safety and effectiveness of the combination of fludarabine plus cyclophosphamide in treating severe aplastic anemia that has not responded to initial treatments.
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia Neutropenia Pancytopenia Severe Aplastic Anemia||Drug: Cyclophosphamide Drug: Fludarabine||Phase 1 Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia|
|Study Start Date :||August 2010|
|Primary Completion Date :||July 2012|
|Study Completion Date :||July 2012|
U.S. FDA Resources
Experimental: Flu/Cy Response at 6 months
The primary objective is to assess Fludarabine/ Cyclophosphamide (Flu/Cy) hematological response in SAA.The primary endpoint will be response at six months.
Other Name: CyDrug: Fludarabine
125 mg/m squared
Other Name: Flu
- Response Rate at 6 Months [ Time Frame: 6 months ]The primary objective is to assess the Flu/Cy hematological response in SAA.The primary endpoint will be response at six months.
- Secondary Endpoints Will Evaluated for the Study to Include: (a) Hematologic Response at 3 and 12 Months and Yearly Thereafter; (b) Relapse (c) Clonal Evolution to Paroxysmal Nocturnal Hemoglobinuria (PNH), Myelodysplasia or Acute Leukemia; (e) Survival. [ Time Frame: 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01187017
|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)|