Find NHLBI Clinical Trials
Search selected NHLBI-supported ClinicalTrials.gov.and by condition, location, or age group. You can also view the complete list of NHLBI-funded studies at
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- Alpha-1 Antitrypsin Deficiency
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Showing 1 - 10 out of 10 results
Sirolimus (Rapamune) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Are you or your child taking cyclosporine for aplastic anemia? This study is testing whether the medicine sirolimus helps prevent the return of aplastic anemia after stopping cyclosporine. Participants in this study must be 2 years or older and have severe aplastic anemia. This study is located in Bethesda, Maryland.
Have you or your child been diagnosed with aplastic anemia but not been treated yet? This study is testing whether adding the medicine eltrombopag to standard treatments is a better way to treat severe aplastic anemia. Participants in this study must be 2 years or older and have severe aplastic anemia that has not yet been treated. This study is located in Bethesda, Maryland.
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Are you considering a blood stem cell transplant? This study is investigating a new method for collecting blood stem cells from donors to see if it reduces transplant complications, such as rejection, in patients who have blood diseases. To participate in this study, you must have a well-matched donor; you and your donor must be between 4 and 80 years old; and you must not be a candidate for immunosuppressive therapy. This study is located in Bethesda, Maryland.
Do you or a family member have Diamond-Blackfan anemia? This registry seeks to collect information from patients who have Diamond-Blackfan anemia (DBA), in order to help researchers and doctors learn more about the condition. The registry will also help connect patients with DBA to research and clinical studies, and possible treatment options. The registry accepts all patients diagnosed with DBA.
Do you have short telomeres and a telomere gene mutation? Some people who have aplastic anemia have very short telomeres, which protect the ends of DNA in chromosomes. This study is testing whether low doses of the medicine danazol help prevent telomeres from getting shorter and reduce signs of damage from aplastic anemia or related conditions. Participants in this study must be 3 years or older and have a telomere disease and signs of aplastic anemia, lung disease such as pulmonary fibrosis, or liver disease. This study is located in Bethesda, Maryland.
Have you received a stem cell transplant? This study aims to monitor the long-term health of patients who have received a donor stem cell transplant from the NIH Clinical Center. To participate in this study, you must be between 10 and 80 years old and had your transplant more than 3 years ago. This study is located in Bethesda, Maryland.
Accepting Healthy Volunteers
Are you scheduled for a stem cell transplant with cord blood? This study will assess the safety and effectiveness of certain cord blood transplants. The study will help researchers learn the best methods for collecting, storing, and using cord blood in transplants. To participate in this study, you must have a disorder that compromises your body’s ability to make blood cells. This study is located in Bethesda, Maryland.
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Do you or your child have severe aplastic anemia? This study tests the safety and effectiveness of two medicines called cyclosporine and eltrombopag in people with severe aplastic anemia. The typical treatment is a combination of three medicines: cyclosporine, eltrombopag, and horse anti-thymocyte globulin. Researchers want to see if starting people at a lower dose of cyclosporine and eltrombopag before giving them horse anti-thymocyte globulin is helpful. Participants in this study must be 4 years or older with severe aplastic anemia. This study is located at the NIH Clinical Center in Bethesda, Maryland.
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Do you have severe aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria and a relative who is willing to donate blood plasma? This study is exploring whether these conditions can be treated using peripheral blood hematopoietic stem cells, which are easier to collect than bone marrow cells. To participate, you must be between 4 and 55 years old with severe aplastic anemia, myelodysplastic syndrome or paroxysmal nocturnal hemoglobinuria and have a relative who is between 4 and 75 years old who can donate peripheral blood stem cells. This study is located at the NIH Clinical Center in Bethesda, Maryland.
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Do you or your child have severe aplastic anemia or myelodysplastic syndrome? This study aims to find new ways to make stem cell transplants safer and more effective. Researchers are testing if treating people with severe aplastic anemia with a co-infusion of blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor is safe and effective. To participate in this study, you must be between 4 and 75 years old. This study is located at the NIH Clinical Center in Bethesda, Maryland.