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Found 23 clinical trials
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Background Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood

cell counts
  • 25 May, 2021
  • 1 location
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.

  • 14 Mar, 2021
  • 1 location
The Use of Trifluoperazine in Transfusion Dependent DBA

Diamond Blackfan anemia (DBA) is a rare inherited pure red cell aplasia. The two main non-stem cell transplant therapeutic options are corticosteroids and red blood cell (RBC) transfusions

antipsychotic agent
pure red cell aplasia
ribosomal protein
  • 11 Mar, 2021
  • 2 locations
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT). The procedure, however, is limited to children younger than the age of 16 because the risks are lower for children than for adults. The purpose of this study is to …

vaso-occlusive crisis
bone marrow transplant
iron overload
conjugated bilirubin
  • 26 Jul, 2021
  • 1 location
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will …

red blood cell transfusions
conditioning regimen
bone marrow transplant
iron overload
blood transfusion
  • 16 May, 2021
  • 2 locations
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

ejection fraction
diamond-blackfan anemia
  • 04 Jun, 2021
  • 1 location
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for …

gilbert's syndrome
t-cell depletion
metabolic disorders
  • 19 Jul, 2021
  • 1 location
Fludarabine Phosphate Cyclophosphamide Total Body Irradiation and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the …

chemotherapy regimen
kostmann's syndrome
waldenstrom's macroglobulinemia
chronic myeloid leukemia
cytogenetic abnormalities
  • 24 Jan, 2021
  • 1 location
Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine A and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also …

  • 17 Jul, 2021
  • 1 location
Diamond Blackfan Anemia Registry (DBAR)

The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA).

white blood cell count
platelet count
  • 16 Mar, 2021
  • 1 location