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  Patient Recruitment - Aplastic Anemia
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Research Studies        
A pilot study of fludarabine plus cyclophosphamide in refractory severe aplastic anemia   Eltrombopag in Moderate Aplastic Anemia Patients   Stem Cell Transplant for Patients with Aplastic Anemia  
Alemtuzumab (Campath) for Refractory or Relapsed Severe Aplastic Anemia   h-ATG for SAA unresponsive to or following a suboptimal response to r-ATG/cyclosporine   Umbilical Cord Transplant to Treat Severe Aplastic Anemia
Eltrombopag in Aplastic Anemia patients with low platelets   Randomized Trial of Campath versus r-ATG/CsA for Severe Aplastic Anemia  

Aplastic Anemia Research Study

A pilot study of fludarabine plus cyclophosphamide in refractory severe aplastic anemia

Hematologists at the National Heart, Lung, and Blood Institute are investigating whether the combination of cyclophosphamide plus fludarabine may help increase blood counts, reduce anemia symptoms, and/or reduce dependence on transfusions in severe aplastic anemia patients who have failed horse ATG. If eligible to participate, consenting subjects will receive an intravenous infusion of study medication cyclophosphamide for 2 days and fludarabine for 5 days. Subjects will be admitted to the NIH Clinical Center hospital for study drug initiation. If the study drug infusion is tolerated well, the subject may be discharged and receive the remainder of the treatment course as an outpatient.

If you have been diagnosed with SAA and have received prior immunosuppressive therapy with horse ATG and have failed to respond, you may be able to participate in this clinical trial. To find out if you qualify, please call our study coordinator at 301-496-4462 or email us at BloodStudy@nhlbi.nih.gov

 
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Aplastic Anemia Research Study

Alemtuzumab (Campath) for Refractory or Relapsed Severe Aplastic Anemia

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for Severe Aplastic Anemia (SAA). The study drug, a monoclonal antibody, may help increase blood counts, reduce anemia symptoms, and/or reduce dependence on transfusions. Subjects will receive an intravenous infusion of study medication alemtuzumab (Campath) once a day for 10 days. Subjects will be admitted to the NIH Clinical Center hospital for study drug initiation. If the study drug infusion is tolerated well, the subject may be discharged and receive the remainder of the treatment course as an outpatient.

If you have been diagnosed with SAA and have received prior immunosuppressive therapy and have either failed to respond or have relapsed following treatment, you may be able to participate in this clinical trial. To find out if you qualify, please call our study coordinator at 301-496-4462 or email us at BloodStudy@nhlbi.nih.gov

 
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Aplastic Anemia Research Study

Eltrombopag in Aplastic Anemia patients with low platelets

If you have been diagnosed with aplastic anemia, are 18 years old or older, and have a low platelet count, you may be able to participate in a clinical trial evaluating eltrombopag, a new FDA-approved drug that has been shown to help the bone marrow produce more platelets in patients with another disorder that causes a low platelet count, immune thrombocytopenia (ITP). We are studying whether or not this drug can increase the platelet count and/or reduce the need for platelet transfusions in patients with aplastic anemia.
If you qualify for study participation, you will take eltrombopag pills once a day for three months. While you are taking eltrombopag and for 30 days after you discontinue the drug, we will monitor you closely for side effects. You will need to be seen at least once a month at the NIH, and weekly for blood count monitoring either with your home physician or at the NIH. There is no charge for the study drug, laboratory tests, examinations, procedures, and/or clinic visits performed as part of this study. Provisions for local travel will be consistent with NIH guidelines. For more information, please contact the research nurse at 301-402-2837
or email us at BloodStudy@nhlbi.nih.gov

 
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Aplastic Anemia Research Study

Eltrombopag in Moderate Aplastic Anemia Patients

This research is investigating whether eltrombopag (Promacta) can be used to treat thrombocytopenia (low platelet counts) or anemia in patients with moderate aplastic anemia. Eltrombopag has been approved by the FDA to treat low blood platelet counts in adults with chronic immune thrombocytopenic purpura. Participants will take Eltrombopag pills daily and will be monitored monthly and for 30 days after the drug is stopped.

There is no charge for study-related medications, laboratory tests, examinations, procedures, and/or clinic visits at the NIH performed as part of this study. Provisions for lodging and local travel, consistent with NIH guidelines, will be provided.
For questions about this study, please contact the research coordinator at : ronan.desmond@nhlbi.nih.gov or at : 301-451-7143




 
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Aplastic Anemia Research Study

h-ATG for SAA unresponsive to or following a suboptimal response to r-ATG/cyclosporine

Hematologists at the National Heart, Lung, and Blood Institute are investigating whether horse anti-thymocyte globulin (h-ATG) may help increase blood counts, reduce anemia symptoms, and/or reduce dependence on transfusions in severe aplastic anemia (SAA) patients who have failed rabbit-ATG. If eligible to participate, consenting subjects will receive an intravenous infusion of study medication h-ATG for 4 days followed by 6 months of oral cyclosporine. Subjects will be admitted to the NIH Clinical Center hospital for study drug initiation. If the study drug infusion is tolerated well, the subject may be discharged and receive the remainder of the treatment course as an outpatient.
If you have been diagnosed with SAA and have received prior immunosuppressive therapy with rabbit-ATG and have either failed to respond or have relapsed following treatment, you may be able to participate in this clinical trial. To find out if you qualify, please call our study coordinator at 301-496-4462 or email us at
BloodStudy@nhlbi.nih.gov
 
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Aplastic Anemia Research Study

Randomized Trial of Campath versus r-ATG/CsA for Severe Aplastic Anemia

Researchers at the National Heart, Lung, and Blood Institute of the National Institutes of Health, a part of the Department of Health and Human Services, are investigating a new method of treatment for severe aplastic anemia. Hematologists are comparing the effectiveness of a new immunosuppressive treatment (Campath-1H) with rabbit ATG and Cyclosporine (CsA) therapy for patients with severe aplastic anemia. The study may help increase blood counts, reduce anemia symptoms, and/or reduce dependence on immunosuppressive medications and transfusions.

If you are determined eligible to participate, you will be selected by a process based on chance to receive either (1) Campath-1H for 10 days or (2) rabbit ATG for 5 days + CsA for 6 months.

If you have been diagnosed with severe aplastic anemia and are ages 2 or older , you may be able to participate in this clinical trial. To find out if you qualify, please contact our coordinator at (301) 496-4462 or email us at BloodStudy@nhlbi.nih.gov

 
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Aplastic Anemia Research Study

Stem Cell Transplant for Patients with Aplastic Anemia

If you have been diagnosed with aplastic anemia or paroxysmal nocturnal hemoglobinuria (PNH), you may be able to participate in a stem cell transplant clinical trial designed to evaluate methods to decrease graft-versus-host disease, promote engraftment, and improve immune system recovery following a bone marrow stem cell transplant procedure.

You must have an HLA-matched family member to participate. You will be given chemotherapy followed by a transfusion of stem cells and lymphocytes from your donor, which will replace your immune system with the immune system of your healthy donor. Post-transplant therapy is designed to reduce the risk of graft-versus-host disease.

We do the blood testing free of charge to see if your family member is a match. We pay for all medical costs related to the transplant procedure. You must be available to live near NIH for approximately 3 months. We also provide a daily allowance to help with living expenses while you are on the study and living away from home.

To inquire about eligibility, contact our research coordinator (301) 594-8013 or by e-mail: BloodStudy@nhlbi.nih.gov

 
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Aplastic Anemia Research Study

Umbilical Cord Transplant to Treat Severe Aplastic Anemia

If you (or someone you know) has been diagnosed with aplastic anemia, you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective for a wide number of diseases and disorders. In this study, we will evaluate the safety and effectiveness using a different type of stem cell transplant procedure, specifically, co-infusion of peripheral blood stem cells from a family member and cord blood stem cells from an unrelated donor.

You must have family member who is willing to participate as a stem cell donor as well as the availability of an HLA matched umbilical cord blood unit (the NIH will conduct the search and procurement) in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 594-8013 or by email at BloodStudy@nhlbi.nih.gov

 
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