Phase 2 N=11 Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
Anemia, Aplastic · Red-Cell Aplasia, Pure · Anemia, Diamond-Blackfan
Primary: Response to Rituximab — 0; 2; 9 participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=15 Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Anemia, Diamond-Blackfan
Primary: Participants That Responded to Eltrombopag — 1; 14 Participants
Phase 2 N=55 Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
Diamond Blackfan Anemia · Blackfan Diamond Syndrome · DBA
Primary: Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia — 2; 5; 36; 0 Participants
Phase 2 N=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Phase 2 N=102 Darbepoetin Administration to Preterm Infants
Infant, Newborn
Primary: Number of Transfusions During Hospitalization — 1.2; 1.2; 2.4 number of transfusions
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=14 Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis
Anemia · Primary Myelofibrosis · Secondary Myelofibrosis
Primary: Best Overall Response Rate as Determined by International Working Group Criteria — 28.6 percentage of patients with CR, PR or CI
Phase 2 N=34 Eltrombopag for Moderate Aplastic Anemia
Moderate Aplastic Anemia · Unilineage Bone Marrow Failure Disorders
Primary: Proportion of Drug Responders — 17 Participants
Phase 2 N=45 Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
Aplastic Anemia · Leukemia · Myelodysplastic Syndrome
Primary: Successful Neutrophil Engraftment — 44; 1 Participants
Phase 2 N=30 Sirolimus for Autoimmune Disease of Blood Cells
Autoimmune Pancytopenia · Autoimmune Lymphoproliferative Syndrome (ALPS) · Evans Syndrome
Primary: Number of Participants With Grade 3 and 4 Toxicities of Administration of Oral Sirolimus — 1 participants
Phase 3 N=147 Darbepoetin Alfa in Patients With Anemic Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
MDS
Primary: Percentage of Participants With at Least One Red Blood Cell (RBC) Transfusion During the Double-blind Treatment Period — 59.2; 36.1 percentage of participants — p=0.008
Phase 2 N=43 A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Anemia, Aplastic · Anemia, Hypoplastic · Thrombocytopenia
Primary: The Portion of Drug Responders as Defined by Hematologic Improvements — 17 Participants
Phase 2 N=14 Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 1; 0; 0; 0 Participants
Phase 2 N=31 Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Fanconi Anemia
Primary: Number of Participants Experiencing Graft Failure — 0; 0; 0 Participants
Phase 2 N=1 Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
Fanconi Anemia
Primary: Number of Participants Who Safely Received AMD3100 Used in Combination With Standard Dose G-CSF — 1 participants
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
N/A N=441 Anemia Management in Chronic Kidney Disease Not on Dialysis Patients After the European Renal Best Practice (ERBP) Working Group Recommendations
ANEMIA
Primary: % Patients Achieving Target Hemoglobin Levels — 42.5; 35.3 percentage of participants
Phase 2 N=47 Alemtuzumab to Treat Severe Aplastic Anemia
Severe Aplastic Anemia, Refractory · Severe Aplastic Anemia, Relapse
Primary: Number of Participants With Hematological Response at 6 Months — 0; 18 Participants
Phase 2 N=27 Danazol for Genetic Bone Marrow and Lung Disorders
Aplastic Anemia
Primary: Number of Patients Having Attenuation of Accelerated Telomere Attrition — 12 Participants
Phase 2 N=12 Efficacy of Eltrombopag to Improve Thrombocytopenia of MYH9-related Disease
Blood Platelet Disorders
Primary: Response to Drug Based on Platelet Count at the End of Therapy — 91.6 percentage of participants
Phase 2 N=48 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Myelodysplastic Syndrome · Aplastic Anemia
Primary: Overall Response — 8; 8; 4 participants
Phase 2 N=23 Hypomethylating Properties of Freeze-dried Black Raspberries (BRB) in Patients With Myelodysplastic Syndrome or Myelodysplastic Syndrome/Myeloproliferative Neoplasm (MDS/MPN)
Myelodysplastic Syndromes
Primary: The Number of Patients With Grade 3 or Higher Serious Adverse Events. — 3 Participants
Phase 2 N=54 Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia
Aplastic Anemia
Primary: Participants no Longer Meeting Criteria for Severe Aplastic Anemia. — 18; 17; 9; 9 participants
Phase 2 N=25 Defibrotide TMA Prophylaxis Pilot Trial
Thrombotic Microangiopathies
Primary: Percent of Total Doses of Defibrotide That Were Missed [Feasibility] — 0.7 percentage of missed doses
Phase 2 N=35 Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia
Aplastic Anemia
Primary: Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline) — 1; 11; 18; 3 Participants
Phase 2 N=136 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
Phase 2 N=20 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months — 9 participants
Phase 2 N=14 Pilot Study of Metformin for Patients With Fanconi Anemia
Fanconi Anemia
Primary: Hematologic Response — 4 Participants
Phase 2 N=6 Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia
Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
Early Phase 1 N=5 RHD Genotype Matched Red Cells for Anti-D
Sickle Cell Disease · Anti-D Antibodies
Primary: Anti-D Recurrence — 0; 5 Participants