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Home › Medications › busulfan

busulfan

3 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials of busulfan

103 trials tracked of busulfan: 9 in phase 3 or 4 and 1 with published results. The most-cited published study has 102 citations.

103Trials tracked
9Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 4 1 Phase 3 8 Phase 2 83 Phase 1 5 Other / NA 6
  1. Phase 3 A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma Completed · 102 cited
  2. Phase 4 Busulfan Safety/Efficacy as Conditioning Prior to Hematopoietic Cell Transplantation (HCT) Completed
  3. Phase 3 Allogeneic Transplantation For Severe Osteopetrosis Completed
  4. Phase 3 IV Busulfan With Allo-BMT: Study for Patients With Acute Myelogenous Leukemia and Myelodysplastic Syndrome Completed
  5. Phase 3 Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer Completed
  6. Phase 3 Fludarabine-IV Busulfan ± Clofarabine and Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) Completed
Show 44 more trials
  1. Phase 3 Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia Completed
  2. Phase 3 Combination Chemotherapy With or Without Monoclonal Antibody Therapy in Treating Patients With AML Leukemia Completed
  3. Phase 3 Stem Cell Transplant for Bone Marrow Failure Syndromes Completed
  4. Phase 2 Pilot Study of Leuprolide to Improve Immune Function After Allogeneic Bone Marrow Transplantation Completed
  5. Phase 2 Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer Completed
  6. Phase 2 Autologous and Allogeneic Transplant for Relapsed Lymphoma Completed
  7. Phase 2 A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies Completed
  8. Phase 2 Combination Chemo, Peripheral Stem Cell Transplant, Biological Therapy, Pamidronate and Thalidomide for Multiple Myeloma Completed
  9. Phase 2 Combination Chemotherapy and Peripheral Blood Stem Cell Transplant Followed By Aldesleukin and Sargramostim in Treating Patients With Inflammatory Stage IIIB or Metastatic Stage IV Breast Cancer Completed
  10. Phase 2 Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen Completed
  11. Phase 2 Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome Completed
  12. Phase 2 Busulfan, Melphalan, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma Completed
  13. Phase 2 Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia Completed
  14. Phase 2 Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders Completed
  15. Phase 2 A Research Study of Bone Marrow Transplantation From Unrelated or Partially Matched Related Donors Completed
  16. Phase 2 Busulfan, Cyclophosphamide, and Autologous Stem Cell Transplant in Treating Patients With Multiple Myeloma Completed
  17. Phase 2 Busulfan, Melphalan, and Thiotepa in Treating Patients Who Are Undergoing an Autologous Stem Cell Transplant for Hodgkin's or Non-Hodgkin's Lymphoma Completed
  18. Phase 2 Busulfan and Fludarabine in Patients With AML and MDS Completed
  19. Phase 2 Once Daily Targeted Intravenous (IV) Busulfex as Part of Reduced-toxicity Conditioning for Patients With Refractory Lymphomas Undergoing Allogeneic Transplantation Completed
  20. Phase 2 Busulfan and Fludarabine Followed by Post-transplant Cyclophosphamide Completed
  21. Phase 2 Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer Completed
  22. Phase 2 Decitabine and Total-Body Irradiation Followed By Donor Bone Marrow Transplant and Cyclophosphamide in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia Completed
  23. Phase 2 Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation Completed
  24. Phase 2 High-Dose Gemcitabine, Busulfan and Melphalan for Patients With Refactory Hodgkin's Disease Completed
  25. Phase 2 Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene Completed
  26. Phase 2 Aza-SAHA-GBM With AutoSCT for Refractory Lymphoma Completed
  27. Phase 2 Azacitidine After Allo Blood And Marrow Transplantation (BMT) for Chronic Myelogenous Leukemia (CML) Completed
  28. Phase 2 Donor Peripheral Stem Cell Transplant, Fludarabine, and Busulfan in Treating Patients With Hematologic Cancers Completed
  29. Phase 2 Busulfan, Fludarabine, Clofarabine With Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia Completed
  30. Phase 2 Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency Completed
  31. Phase 2 High Dose Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil in Preventing Graft Versus Host Disease in Patients With Hematological Malignancies Undergoing Myeloablative or Reduced Intensity Donor Stem Cell Transplant Completed
  32. Phase 2 Combination Chemotherapy, Bone Marrow Transplant, and Post Transplant Cyclophosphamide for Hematologic Cancer Completed
  33. Phase 2 Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies Completed
  34. Phase 2 Donor Natural Killer Cells and Donor Stem Cell Transplant in Treating Patients With High Risk Myeloid Malignancies Completed
  35. Phase 2 Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplant Completed
  36. Phase 2 Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia Completed
  37. Phase 2 Therapy for Children With Advanced Stage Neuroblastoma Completed
  38. Phase 2 Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer Completed
  39. Phase 2 Thiotepa-Clofarabine-Busulfan With Allogeneic Stem Cell Transplant for High Risk Malignancies Completed
  40. Phase 2 Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease Completed
  41. Phase 2 Blood Stem Cell Transplantation for the Treatment of Older Patients With Acute Myelogenous Leukemia Completed
  42. Phase 2 Phase I/II Trial of Fludarabine Plus Busulfan and Allogeneic Progenitor Cell Support Completed
  43. Phase 2 Busulfan, Fludarabine Phosphate, and Anti-Thymocyte Globulin Followed By Donor Stem Cell Transplant and Azacitidine in Treating Patients With High-Risk Myelodysplastic Syndrome and Older Patients With Acute Myeloid Leukemia Completed
  44. Phase 2 Cyclophosphamide for Prevention of Graft-Versus-Host Disease After Allogeneic Peripheral Blood Stem Cell Transplantation in Patients With Hematological Malignancies Completed

Showing the 50 most-cited and recently-updated of 103 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.
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Autologous hematopoietic stem cell transplantation improves disease-free survival in pediatric extraocular retinoblastoma patients
Pediatrics Cohort
Autologous hematopoietic stem cell transplantation improves disease-free survival in pediatric extraocular retinoblastoma patients Children With Eye Cancer Now Have a New Hope for Survival
This retrospective analysis included 12 children with extraocular retinoblastoma treated with autologous hematopoietic stem cell transplanta…
Children with advanced eye cancer can now achieve long-term survival using their own stem cells, especially if the disease has not reached t…
Frontiers Apr 29, 2026
Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia
Genetics & Precision Medicine Phase I
Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia New Gene Edit Stops Blood Transfusions
A single-group phase 1-2 trial in 9 adults with transfusion-dependent β-thalassemia found all participants achieved neutrophil engraftment a…
A new gene edit reactivates natural red blood cell production, potentially ending the need for lifelong transfusions in adults with severe b…
Apr 11, 2026
Renizgamglogene autotemcel infusion showed engraftment and hemoglobin increases in severe sickle cell disease patients
Hematology Phase I
Renizgamglogene autotemcel infusion showed engraftment and hemoglobin increases in severe sickle cell disease patients One Gene Edit Stopped Painful Sickle Cell Crises in Most Patients
This single-group, open-label, multicenter Phase 1-2 trial enrolled 28 patients with severe sickle cell disease aged 12 to 50 years.
A single infusion of CRISPR-edited stem cells eliminated painful sickle cell crises in 27 out of 28 patients by switching the body back to p…
Apr 10, 2026