Phase 2 N=136 Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
Immunosuppresion · Thrombocytopenia · Pancytopenia
Primary: Hematologic Response — 38; 20; 3 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=22 Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Blood Counts and Adverse Event Profile After 6 Months of Treatment. — 4; 5; 12 participants
Phase 2 N=21 NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
Severe Aplastic Anemia · Aplastic Anemia · Bone Marrow Failure
Primary: Overall Survival and Engraftment at One Year — 19 Participants
N/A N=4 Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Anemia, Aplastic · Amegakaryocytic Thrombocytopenia · Diamond-Blackfan Anemia
Primary: Treatment Failures
Phase 2 N=31 Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA) · Severe Aplastic Anemia (SAA)
Primary: Number of Participants Who Engrafted by Day 42 — 30 Participants
Phase 2 N=1 A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
Aplastic Anemia · Neutropenia · Pancytopenia
Primary: Response Rate at 6 Months — 1; 0; 0 participants
Phase 2 N=18 Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Severe Aplastic Anemia · Bone Marrow Failure Syndromes
Primary: Is This Type of Transplantation for Severe Aplastic Anemia Feasible and Safe? — 18 Participants
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=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
N/A N=9 Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Number of Patients With Engraftment Response — 9 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=32 Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)
Severe Aplastic Anemia
Primary: Percentage of Participants With Overall Survival (OS) — 80.6 percentage of participants
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 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 Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment
Anemia, Aplastic · Anemia, Hypoplastic
Primary: Number of Participants With Complete Response at 3 Months. — 0 Participants
Phase 2 N=36 Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
Severe Aplastic Anemia (SAA)
Primary: Complete Response (CR) Rate at Week 26 — 16.7; 25; 15.4; 0 percentage of participants
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
Phase 2 N=35 A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
Aplastic Anemia
Primary: Proportion of Subjects Achieving a Platelet Response at Week 9 — 0; 0; 3; 7 Participants
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=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=34 Eltrombopag for Moderate Aplastic Anemia
Moderate Aplastic Anemia · Unilineage Bone Marrow Failure Disorders
Primary: Proportion of Drug Responders — 17 Participants
Phase 2 N=97 Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)
Anemia, Aplastic
Primary: Disease-free Survival (DFS) — 6; 3; 9; 4 participants
Phase 2 N=21 Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant
Aplastic Anemia
Primary: Incidence of Chronic GVHD — 5 number participants with chronic GVHD
Phase 2 N=13 Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
Severe Aplastic Anemia · Very Severe Aplastic Anemia · Moderate Aplastic Anemia
Primary: Proportion of Participants With Platelet Response — 0.23 proportion of participants
Phase 2 N=10 Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects
Aplastic Anemia
Primary: ORR at 6 Months: Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at Week…
Phase 2 N=54 Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
Severe Aplastic Anemia
Primary: Overall Hematologic Response Rate by 6 Months — 46.3 Percentage of participants
Phase 2 N=20 Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
Aplastic Anemia
Primary: Hematologic Response Rate at 6 Months (Week 26) by Investigator — 70.0 Percentage of 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=40 Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Severe Aplastic Anemia (SAA)
Primary: Number of Participants With Drug Response as Defined by Clinically-signficant Hematologic Improvements — 20 Participants