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Severe aplastic anemia

Part of Anemia

4 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for severe aplastic anemia

35 trials tracked for severe aplastic anemia: 2 in phase 3 or 4 and 2 with published results. The most-cited published study has 151 citations.

35Trials tracked
2Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 3 2 Phase 2 28 Other / NA 5
  1. Phase 3 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA Completed · 151 cited
  2. Phase 3 A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia Completed
  3. Phase 2 A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia Completed
  4. Phase 2 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 Completed
  5. Phase 2 A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies Completed
  6. Phase 2 NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders Completed
Show 29 more trials
  1. Phase 2 Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia Completed
  2. Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  3. Phase 2 Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Completed
  4. Phase 2 Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia Completed
  5. Phase 2 Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects Completed
  6. Phase 2 Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Completed
  7. Phase 2 Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients Completed
  8. Phase 2 Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Completed
  9. Phase 2 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome Completed
  10. Phase 2 A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Completed
  11. Phase 2 Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies Completed
  12. Phase 2 Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia Completed
  13. Phase 2 Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies Completed
  14. Phase 2 NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Completed
  15. Phase 2 Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects. Completed
  16. Phase 2 Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502) Completed
  17. Phase 2 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 Completed
  18. Phase 2 Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor Completed
  19. Phase 2 Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  20. Phase 2 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia Completed
  21. Phase 2 Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia Completed
  22. Phase 2 Eltrombopag for Moderate Aplastic Anemia Completed
  23. Phase 2 Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia Completed
  24. Phase 2 Danazol for Genetic Bone Marrow and Lung Disorders Completed
  25. N/A Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia Completed
  26. N/A Unrelated Umbilical Cord Blood (UBC)Transplantation Completed
  27. N/A Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders Completed
  28. N/A Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia Completed
  29. N/A Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies Completed

Showing the 35 most-cited and recently-updated of 35 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.

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Questions about severe aplastic anemia

What clonal evolution patterns are seen in severe aplastic anemia treated with eltrombopag and immunosuppression?

Treatment with eltrombopag and immunosuppression leads to an increase in clonal mutations over time, with two main patterns of evolution: early changes involving chromosome 7 and late changes driven by pre-existing clones that can progress to cancer or PNH.

Full answer →