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Research Studies        
A pilot study of fludarabine plus cyclophosphamide in refractory severe aplastic anemia   Ofatumumab for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma   Eltrombopag in Myelodysplastic syndrome (MDS) patients with low platelets  
Alemtuzumab (Campath) for Refractory or Relapsed Severe Aplastic Anemia   Partially Matched DLI to Treat Relapsed Leukemia   Escalating Doses of ON 01910.Na for Myelodysplastic Syndrome (MDS)  
Eltrombopag in Aplastic Anemia patients with low platelets   PCI-32765 for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in patients 65 & older or have a 17p deletion   Non-myeloablative stem cell transplant for patients with myelodysplastic syndrome  
Eltrombopag in Moderate Aplastic Anemia Patients   Stem Cell Transplant and T Cell Add Back to Treat Leukemia   Partially Matched DLI to Treat Relapsed MDS  
h-ATG for SAA unresponsive to or following a suboptimal response to r-ATG/cyclosporine   Alemtuzumab (Campath) for T-LGL Lymphoproliferative Disorders   Stem Cell Transplant to Treat Myelodysplastic Syndrome (MDS)  
Randomized Trial of Campath versus r-ATG/CsA for Severe Aplastic Anemia   Escalating Doses of ON 01910.Na for Lymphoma   Umbilical Cord Transplant to Treat Myelodysplastic Syndrome (MDS)  
Stem Cell Transplant for Patients with Aplastic Anemia   HLA-matched Mobilized Peripheral Blood Hematopoietic Stem Cell Transplantation for Advanced Mycosis Fungoides/Sezary Syndrome   Stem Cell Transplant as Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH)  
Umbilical Cord Transplant to Treat Severe Aplastic Anemia   Ofatumumab for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma   Collection and Storage of Umbilical Cord Blood for Sickle Cell Disease  
Autologous NK cell infusions in treatment resistant Cancer   Stem Cell Transplant to Treat Lymphoma   Natural History of Sickle Cell Disease  
Stem Cell Transplant for Treatment of Metastatic Renal Cell (Kidney) Cancer   Stem Cell Transplant for High Risk Patients with Lymphoma   Pulmonary Hypertension Research Screening Study  
Stem Cell Transplant for Diamond Blackfan Anemia   Escalating Doses of ON 01910.Na for Multiple Myeloma   PUSH - Risk Factors for Pulmonary Hypertension  
Autologous NK cell infusions in treatment resistant CLL and CML   Stem Cell Transplant to Treat Multiple Myeloma   Stem Cell Transplant for Sickle Cell Anemia  
Escalating Doses of ON 01910.Na for Leukemia   HLA-matched Mobilized Peripheral Blood Hematopoietic Stem Cell Transplantation for Advanced Mycosis Fungoides/Sezary Syndrome   Stem Cell Transplant for Thalassemia
Natural History of MBL, CLL and SLL   Alemtuzumab (Campath-1H) for Myelodysplastic Syndrome (MDS)  

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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Cancer Research Study

Autologous NK cell infusions in treatment resistant Cancer

If you (or someone you know) has metastatic cancer of the lung, prostate, colon, rectum, kidney, pancreas or malignant melanoma that is not responding to standard treatments, you may be able to participate in our clinical trial designed to evaluate the safety of a new type of cell therapy. Specifically, we are testing the safety and tolerability of the infusion of a type of your own white blood cells called NK cells.

In this research project, your NK cells will be collected, reproduced to large numbers in the laboratory and then be given back to you as an infusion. Before the cells are given back, you will receive two chemotherapy agents: pentostatin, to suppress your immune system and prevent it from attacking the infused NK cells, and bortezomib, to increase the anti-tumor activity of the NK cells. You will also receive the drug interleukin-2 to maintain NK cell activity. You are required to return to the Clinical Center for frequent safety monitoring over a 7 week period of protocol participation. If you respond you may have the option to continue NK cell infusions.

To find out if you qualify, please call us toll free at 1-800-411-1222 (TTY 1-866-411-1010). Our email address is BloodStudy@nhlbi.nih.gov


 
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Cancer Research Study

Stem Cell Transplant for Treatment of Metastatic Renal Cell (Kidney) Cancer

If you have been diagnosed with metastatic renal cell (kidney) cancer, you may be eligible for a stem cell transplant procedure here at the National Institutes of Health (NIH). Under evaluation is whether graft-versus-tumor effects can be generated against renal cell tumors. 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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Diamond Blackfan Anemia / Inherited Bone Marrow Failure Syndromes Research Study

Stem Cell Transplant for Diamond Blackfan Anemia

If you (or someone you know) is between the ages of 16-65 years and have been diagnosed with Diamond Blackfan anemia, you may be eligible for a stem cell transplantation procedure at the Clinical Center of the National Institutes of Health (NIH). Under evaluation is the use of low-dose radiation and novel methods of transplant preparation and post transplant therapy to reduce the risk of graft-versus-host disease.

You must have an HLA-matched family member to participate. We will 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 four months. We also provide a small daily allowance to help with living expenses while you are on the study and living away from home.

For more information please contact our research coordinator at (301) 402-3087, or email us at BloodStudy@nhlbi.nih.gov

 
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Leukemia Research Study

Autologous NK cell infusions in treatment resistant CLL and CML

If you (or someone you know) has CLL or CML that is not responding to standard treatments, you may be able to participate in our phase 1 clinical trial designed to evaluate the safety of a new type of cell transplant. Specifically, we are testing the safety and tolerability of the infusion of one type of your own white blood cells called NK cells.

In this research project your NK cells will be collected, reproduced in large numbers in the laboratory and then given back to you as an infusion. Before the cells are given back, you will receive two chemotherapy agents: pentostatin, to suppress your immune system and prevent it from attacking the infused NK cells, and bortezomib, to increase the anti-tumor activity of the NK cells. You will also receive the drug interleukin-2 to maintain NK cell activity. You are required to return to the Clinical Center for frequent safety monitoring over a 7 week period of protocol participation. If you respond you may have the option to continue NK cell infusions.

To find out if you qualify, please call us toll free at 1-800-411-1222 (TTY 1-866-411-1010). Our email address is BloodStudy@nhlbi.nih.gov

 
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Leukemia Research Study

Escalating Doses of ON 01910.Na for Leukemia

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for chronic lymphocytic leukemia (CLL) and Hairy cell Leukemia. The study drug, ON01910.Na is classified as mitotic inhibitor and works by interrupting the life cycle of leukemia cells. Consenting subjects will receive 2 days of drug infusion once every other week (1 cycle) for a minimum of 2 cycles. Subjects without toxicity will have the option to continue the regimen for 2 additional cycles, after which if there is evidence of response, subjects may continue the regimen indefinitely.

If you are between the ages of 18 and 99 and have been diagnosed with CLL and have failed to respond to, relapsed following, are not eligible for, or opted not to participate in other standard of care treatment options you may be able to participate in a clinical trial. This study will determine what dose and which dosing schedule of this new drug is safe. We will also evaluate whether or not this drug can slow progression of your illness, improve blood counts, and/or reduce the need for transfusions of blood and/or platelets.

To find out if you qualify, please call our study coordinator at 301-402-0797 or email us at BloodStudy@nhlbi.nih.gov
 
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Leukemia Research Study

Natural History of MBL, CLL and SLL

The Hematology Branch of the National Heart, Lung, and Blood Institute is conducting a clinical study for previously untreated patients with Chronic Lymphocytic Leukemia (CLL), Small Lymphocytic Leukemia (SLL) or Monoclonal B cell lymphocytosis (MBL). Although all three diseases are considered a type of blood cancer involving B-lymphocytes, CLL involves the peripheral blood, SLL mostly involves the lymph nodes, and MBL is thought to be the earliest diagnostic stage of CLL/SLL.

Most patients with CLL/SLL/MBL do not require treatment at the time of diagnosis and are observed following a "watch and wait" approach. This study provides state of the art diagnostic workup, regular follow-up monitoring, and sample collection during the "watch and wait" period. If treatment becomes necessary, patients will receive information on possible standard treatment options and research studies available at NIH.

If you choose to participate, you will undergo tests and procedures designed to evaluate the status of your disease. You will also have the opportunity to donate blood and tissue samples that will be used in laboratory research experiments designed to help us better understand MBL/CLL/SLL.

If you or someone you know has been diagnosed with MBL, CLL, or SLL you (they) may be able to participate in this study. To find out if you qualify, please contact our research coordinator at (301) 402-0797 or email us at BloodStudy@nhlbi.nih.gov

 
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Leukemia Research Study

Ofatumumab for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma

The purpose of this study is to evaluate the efficacy of ofatumumab in combination with chemotherapy for previously untreated patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma(SLL). Ofatumumab is FDA approved for treating CLL/SLL that is not responsive to other drugs.

Participants will receive treatment at the NIH Clinical Center Day hospital and will be regularly scheduled for clinic visits at the NIH to evaluate the response to treatment. There is no charge for study-related medications, laboratory tests or examinations. Financial support towards the cost of lodging and travel is available based on NIH guidelines.
For questions about this study, please contact the research coordinator, Tel. No. 301- 402-0797 or BloodStudy@nhlbi.nih.gov

 
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Leukemia Research Study

Partially Matched DLI to Treat Relapsed Leukemia

If you or someone you know is diagnosed with ALL, AML, or CML and have relapsed within 6 months of receiving a stem cell transplant, you may be able to participate in our clinical trial designed to evaluate the effectiveness of a partially HLA matched infusion of donor lymphocytes (DLI). Specifically, we are testing whether giving an infusion of donor lymphocytes from a partially matched family member will maximize the antileukemia effect of following an HLA matched allogeneic stem cell transplant. To achieve at least temporary engraftment of the DLI and disease control, cyclophosphamide and fludarabine immunosuppression will be given prior to the DLI.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov


 
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Leukemia Research Study

PCI-32765 for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in patients 65 & older or have a 17p deletion

This study will investigate the efficacy of PCI-32765 in patients with chronic lymphocytic leukemia (CLL) / small lymphocytic lymphoma (SLL) who are older than 65 or those patients with a 17p deletion. Recent studies indicated that PCI-32765 may inhibit cell proliferation. This protocol is intended both for patients who are untreated and those who have previously been treated. Oral PCI-32765 tablets will be distributed by the NIH and administered at the patient's home. Patients will be followed for a total of 12 months at the NIH. There is no charge for medications, laboratory tests, examinations, procedures and/or clinic visits performed as part of this study. If you have any questions about this study, contact the research nurse, Tel. No. (301)402-0797
 
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Leukemia Research Study

Stem Cell Transplant and T Cell Add Back to Treat Leukemia

If you or someone you know is diagnosed with acute lymphocytic leukemia (ALL), acute myelogenous leukemia (AML), chronic lymphocytic leukemia (CLL), or chronic myrlogenous leukemia (CML), you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective. Specifically, we are testing whether special treatment of donor stem cells and delaying the transfusion of donor immune cells will reduce complications and improve transplant outcome. You must have an HLA-matched brother or sister who is willing to participate as a donor in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov

 
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Lymphoma Research Study

Alemtuzumab (Campath) for T-LGL Lymphoproliferative Disorders

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for T-Large Granular Lymphocyte (T-LGL) Lymphoproliferative Disorders. The study drug, a monoclonal antibody, may help increase blood counts and reduce the number of abnormal T cell large granular lymphocytes. If eligible to participate, consenting 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 T-LGL and are 18 years or older, 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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Lymphoma Research Study

Escalating Doses of ON 01910.Na for Lymphoma

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for Mantle Cell Lymphoma (MCL), Small Lymphocytic Lymphoma (SLL), Prolymphocytic lymphoma (PLL), or Waldenstrom's Macroglobulinemia (also known as lymphoplasmacytic lymphoma or (WM). The study drug, ON01910.Na is classified as mitotic inhibitor and works by interrupting the life cycle of lymphoma cells. Consenting subjects will receive 2 days of drug infusion once every other week (1 cycle) for a minimum of 2 cycles. Subjects without toxicity will have the option to continue the regimen for 2 additional cycles, after which if there is evidence of response, subjects may continue the regimen indefinitely.

If you are between the ages of 18 and 99 and have been diagnosed with MCL, SLL, PLL or WM and have failed to respond to, relapsed following, are not eligible for, or opted not to participate in other standard of care treatment options you may be able to participate in a clinical trial. This study will determine what dose and which dosing schedule of this new drug is safe. We will also evaluate whether or not this drug can slow progression of your illness, improve adenopathy, improve blood counts and/or reduce the need for transfusions of blood and/or platelets.

To find out if you qualify, please call our study coordinator at 301-402-0797 or email us at BloodStudy@nhlbi.nih.gov
 
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Lymphoma Research Study

HLA-matched Mobilized Peripheral Blood Hematopoietic Stem Cell Transplantation for Advanced Mycosis Fungoides/Sezary Syndrome

This research is with the Stem Cell Transplant Program investigating the use of allogeneic (donor) stem cell transplantation in patients with mycosis fungoides/Sezary syndrome who have no reasonable expectation of cure from available therapy. The transplant procedure usually involves the use of chemotherapy and replacement of bone marrow cells from a donor family member. Most of the procedures, including the transplant, are performed in the outpatient setting. Participants will have regularly scheduled follow-up monitoring visits for up to 5 years. Provisions for lodging and local travel will be provided consistent with NIH guidelines. There is no charge for medications, laboratory tests, examinations, procedures and/or clinic visits performed as part of this study.If you have any questions about this study, contact the research Coordinator, Tel. No. 301.594.8013 or BloodStudy@nhlbi.nih.gov

 
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Lymphoma Research Study

Ofatumumab for chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma

The purpose of this study is to evaluate the efficacy of ofatumumab in combination with chemotherapy for previously untreated patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma(SLL). Ofatumumab is FDA approved for treating CLL/SLL that is not responsive to other drugs.

Participants will receive treatment at the NIH Clinical Center Day hospital and will be regularly scheduled for clinic visits at the NIH to evaluate the response to treatment. There is no charge for study-related medications, laboratory tests or examinations. Financial support towards the cost of lodging and travel is available based on NIH guidelines.
For questions about this study, please contact the research coordinator, Tel. No. 301- 402-0797 or BloodStudy@nhlbi.nih.gov

 
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Lymphoma Research Study

Stem Cell Transplant to Treat Lymphoma

If you or someone you know is diagnosed with Non- Hodgkin's lymphoma or Mantle Cell lymphoma, you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective. Specifically, we are testing whether special treatment of donor stem cells and delaying the transfusion of donor immune cells will reduce complications and improve transplant outcome. You must have an HLA-matched brother or sister who is willing to participate as a donor in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov

 
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Lymphoma Research Study

Stem Cell Transplant for High Risk Patients with Lymphoma

If you have been diagnosed with Non-Hodgkin's lymphoma or Hodgkin's Disease 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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Multiple Myeloma Research Study

Escalating Doses of ON 01910.Na for Multiple Myeloma

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for Multiple Myeloma (MM). The study drug, ON01910.Na is classified as mitotic inhibitor and works by interrupting the life cycle of myeloma cells. Consenting subjects will receive 2 days of drug infusion once every other week (1 cycle) for a minimum of 2 cycles. Subjects without toxicity will have the option to continue the regimen for 2 additional cycles, after which if there is evidence of response, subjects may continue the regimen indefinitely.

If you are between the ages of 18 and 99 and have been diagnosed with MM and have failed to respond to, relapsed following, are not eligible for, or opted not to participate in other standard of care treatment options you may be able to participate in a clinical trial. This study will determine what dose and which dosing schedule of this new drug is safe. We will also evaluate whether or not this drug can slow progression of your illness, improve blood counts, and/or reduce the need for transfusions of blood and/or platelets.

To find out if you qualify, please call our study coordinator at 301-402-0797 or email us at BloodStudy@nhlbi.nih.gov
 
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Multiple Myeloma Research Study

Stem Cell Transplant to Treat Multiple Myeloma

If you (or someone you know) have been diagnosed with Multiple Myeloma or Waldenstrom's macroglobulinemia, you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective. Specifically, we are testing whether special treatment of donor stem cells and delaying the transfusion of donor immune cells will reduce complications and improve transplant outcome. You must have an HLA-matched brother or sister who is willing to participate as a donor in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov

 
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Mycosis Fungoides / Sezary Syndrome Research Study

HLA-matched Mobilized Peripheral Blood Hematopoietic Stem Cell Transplantation for Advanced Mycosis Fungoides/Sezary Syndrome

This research is with the Stem Cell Transplant Program investigating the use of allogeneic (donor) stem cell transplantation in patients with mycosis fungoides/Sezary syndrome who have no reasonable expectation of cure from available therapy. The transplant procedure usually involves the use of chemotherapy and replacement of bone marrow cells from a donor family member. Most of the procedures, including the transplant, are performed in the outpatient setting. Participants will have regularly scheduled follow-up monitoring visits for up to 5 years. Provisions for lodging and local travel will be provided consistent with NIH guidelines. There is no charge for medications, laboratory tests, examinations, procedures and/or clinic visits performed as part of this study.If you have any questions about this study, contact the research Coordinator, Tel. No. 301.594.8013 or BloodStudy@nhlbi.nih.gov

 
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Myelodysplastic Syndromes (MDS) Research Study

Alemtuzumab (Campath-1H) for Myelodysplastic Syndrome (MDS)

Hematologists at the National Heart, Lung, and Blood Institute are investigating a new treatment for myelodysplastic syndrome (MDS). The study drug, a monoclonal antibody, may help increase blood counts, reduce anemia symptoms, and/or reduce dependence on transfusions. If eligible to participate, consenting subjects will receive an intravenous infusion of study medication alemtuzumab (Campath-1H) 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 MDS and are 18 years or older, you may be able to participate in this clinical trial. To find out if you qualify, please call our study coordinator at 301-594-4180 or email us at BloodStudy@nhlbi.nih.gov

 
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Myelodysplastic Syndromes (MDS) Research Study

Eltrombopag in Myelodysplastic syndrome (MDS) patients with low platelets

If you have been diagnosed with myelodysplastic syndrome, are18 years old or older, and have a low platelet count, you may be able to participate in a NIH sponsored 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 myelodysplastic syndrome.

If you qualify for study participation, you will take eltrombopag pills by mouth 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 been seen at least once a month at the NIH, and weekly for blood count monitoring either with your home physician or at the NIH. To find out if you qualify or for more information, please contact our study coordinator 301-594-4180 or email us at BloodStudy@nhlbi.nih.gov


 
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Myelodysplastic Syndromes (MDS) Research Study

Escalating Doses of ON 01910.Na for Myelodysplastic Syndrome (MDS)

If you are between the ages of 18 and 85 and have been diagnosed with Myelodysplastic Syndrome (MDS), you may be able to participate in a clinical trial evaluating a new experimental anti-cancer therapy. This study will determine what dose and which dosing schedule of this new drug is safe. We will also evaluate whether or not this drug can slow progression of your illness, improve blood counts, and/or reduce the need for transfusions of blood and/or platelets.

Study participants will receive 3-5 days of drug infusion ( 1 cycle) every other week for a minimum of 2 cycles and a maximum of 8 cycles.

To find out if you qualify or for more information, please contact our study coordinator at 301-594-4180 or email us at BloodStudy@nhlbi.nih.gov


 
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Myelodysplastic Syndromes (MDS) Research Study

Non-myeloablative stem cell transplant for patients with myelodysplastic syndrome

If you have been diagnosed with MDS, 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 at (301) 594-8013 or by e-mail BloodStudy@nhlbi.nih.gov
 
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Myelodysplastic Syndromes (MDS) Research Study

Partially Matched DLI to Treat Relapsed MDS

If you or someone you know is diagnosed with myelodysplastic syndrome (MDS) and have relapsed within 6 months of receiving a stem cell transplant, you may be able to participate in our clinical trial designed to evaluate the effectiveness of a partially HLA matched infusion of donor lymphocytes (DLI). Specifically, we are testing whether giving an infusion of donor lymphocytes from a partially matched family member will maximize the antileukemia effect of following an HLA matched allogeneic stem cell transplant. To achieve at least temporary engraftment of the DLI and disease control, cyclophosphamide and fludarabine immunosuppression will be given prior to the DLI.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov

 
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Myelodysplastic Syndromes (MDS) Research Study

Stem Cell Transplant to Treat Myelodysplastic Syndrome (MDS)

If you (or someone you know) have been diagnosed with Myelodysplastic Syndrome, you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective. Specifically, we are testing whether special treatment of donor stem cells and delaying the transfusion of donor immune cells will reduce complications and improve transplant outcome. You must have an HLA-matched brother or sister who is willing to participate as a donor in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 496-3841 or by email at BloodStudy@nhlbi.nih.gov

 
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Myelodysplastic Syndromes (MDS) Research Study

Umbilical Cord Transplant to Treat Myelodysplastic Syndrome (MDS)

If you (or someone you know) has been diagnosed with myelodysplastic syndrome, 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 a 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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Paroxysmal Nocturnal Hemoglobinuria (PNH) Research Study

Stem Cell Transplant as Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH)

Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) have an abnormal blood stem cell population which produces faulty red blood cells which are prematurely destroyed in the circulation. This protocol is designed to determine whether healthy stem cells can replace abnormal PNH stem cells in patients with severe PNH. Blood stem cells which grow to become mature blood cells will be collected from a tissue compatible family member. The patient with PNH will be given chemotherapy followed by a transfusion of stem cells from their donor. The procedure will replace the patients abnormal or "PNH" stem cells with normal healthy stem cells from the donor.

Patients on this study live in the area for approximately four months. We pay for all medical costs related to the transplant. Patients are responsible for living expenses while here, although we provide some daily financial assistance while on study.

To inquire about eligibility, contact our research coordinator at (301) 594-8013 or by e-mail at BloodStudy@nhlbi.nih.gov
 
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Sickle Cell Anemia Research Study

Collection and Storage of Umbilical Cord Blood for Sickle Cell Disease

If you are pregnant and expecting a baby that may have sickle cell disease you might be interested in our research study. We are collecting umbilical cord blood from newborns that may have sickle cell disease and we are freezing the hematopoietic stem cells that are in the cord blood. Hematopoietic stem cells are used in transplant treatment of sickle cell disease; the more cells the doctors use the better the outcome. The goal of our research is to come up with the best ways to harvest, freeze and thaw the stem cells from the umbilical cord blood so when it comes time to use the stem cells for treatment there will be a lot of high quality cells to use.

To be eligible for this program the pregnant mother must be between the ages of 18 - 45 and she must plan to deliver her infant at a hospital in the Washington Metropolitan Area. The pregnant mother needs to attend a meeting with the nurse coordinator in order to go over the program and give consent before labor begins. There is no cost to the family or their insurance carrier for this program.

For further information please contact the research nurse coordinator at
Office phone: 301-402-2104
Fax: 301-402-3088

 
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Sickle Cell Anemia Research Study

Natural History of Sickle Cell Disease

This research protocol allows us to evaluate and treat you because you have sickle cell disease, sickle cell trait, or another anemia or red blood cell disorder such as thalassemia. This will help us gain more understanding of your disease, painful attacks and lung complications often associated with these anemias. You will receive standard medical care for your blood disease and its complications. This means that you will not receive experimental treatment under this protocol.

Your evaluation may also provide information indicating that you are eligible for another protocol at the NIH. If so the other protocol will be discussed with you. You are under no obligation to participate. This study enrolls individuals ages 5 and over.

Please contact our research study nurse at 301-435-2345 or email VascularStudy@nhlbi.nih.gov
 
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Sickle Cell Anemia Research Study

Pulmonary Hypertension Research Screening Study

Researchers at the NIH invite you to take part in a study that will help us learn how often people with sickle cell anemia develop a complication called pulmonary hypertension. Pulmonary hypertension is a serious disease in which blood pressure in the artery to the lungs is elevated.

You are eligible to participate if you are 18 years of age or older and have sickle cell disease. This study consists of an initial evaluation (including a physical exam, blood tests, and an echocardiogram), a follow-up telephone interview every year after the initial echocardiogram, and a repeat echocardiogram and blood tests approximately two years after the first. There is no cost to participate.

Please contact the study coordinator at 301-402-2104 for more information or email VascularStudy@nhlbi.nih.gov
 
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Sickle Cell Anemia Research Study

PUSH - Risk Factors for Pulmonary Hypertension

The study will look at the risk factors for pulmonary hypertension (high blood pressure in the lungs) in children over 3 years of age and adolescents with sickle cell anemia (SCA) and examine the role of hypoxia (oxygen shortage) in the disease. In patients with SCA, red blood cells become sickle-shaped and tend to form clumps that get stuck in blood vessels, blocking blood flow to the limbs and organs. Blocked blood vessels can cause pain, serious infections, and organ damage. Many patients with SCA also develop pulmonary hypertension.

Children and adolescents with SCA or Chuvash polycythemia (another blood disorder that carries an increased risk for pulmonary hypertension) may be eligible for this study.

Participants undergo the following procedures at the beginning (baseline) and end of the study:
History, physical examination and blood tests .
Echocardiography (ultrasound study of heart function).
Lung function tests.
6-minute walk (measure of the distance covered in 6 minutes of walking).

In addition, patients are followed by telephone or by clinic visits every 6 months for a review of their medical history and medications. A physical examination is also done at 12 months.

BloodStudy@nhlbi.nih.gov
 
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Sickle Cell Anemia Research Study

Stem Cell Transplant for Sickle Cell Anemia

If you (or someone you know) is at least 4 years of age and have severe sickle cell disease, you may be eligible for a stem cell transplantation procedure at the Clinical Center of the National Institutes of Health (NIH). We are studying low-dose radiation and a new combination of medications to improve transplant results.

You must have a healthy first-degree family member (parent, child, or brother or sister) to participate. We pay for all medical costs related to the transplant procedure. You must be available to live near the NIH for approximately four months. A small daily allowance may be provided to help with living expenses while you are on the study and living away from home.

For more information please contact our research coordinator at (301) 402-6466 or by email at BloodStudy@nhlbi.nih.gov
 
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Thalassemia Research Study

Stem Cell Transplant for Thalassemia

Stem Cell Transplantation for Thalassemia

If you (or someone you know) is between the ages of 16-65 years have been diagnosed with thalassemia, you may be eligible for a stem cell transplantation procedure at the Clinical Center of the National Institutes of Health (NIH). Under evaluation is the use of low-dose radiation and novel methods of transplant preparation and post-transplant therapy to reduce the risk of graft-versus-host disease.

You must have an HLA-matched family member to participate. We will 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 four months. We also provide a small daily allowance to help with living expenses while you are on the study and living away from home.

For more information please contact our research coordinator at (301) 402-3087 or by email at BloodStudy@nhlbi.nih.gov

 
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