271 completed, 81 unknown, 74 terminated, 46 recruiting. 372 Phase 2, 133 Phase 1, 78 Phase 3, 47 Not applicable, 7 Phase 4.
Follow Busulfan: new trials by email Literature Review of Busulfan trials Research Report on Busulfan
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) Allogeneic Hematopoietic Stem Cell Transplantation, Biospecimen Collection, Bone Marrow Biopsy - Acute Myeloid Leukemia | Phase 2 | Not yet recruiting | National Cancer Institute (NCI) | 244 | 2027-01-15 | 2026-10-20 |
| CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome Busulfan, Melphalan, Fludarabine - Myelodysplastic Syndromes, Graft Vs Host Disease | Not applicable | Not yet recruiting | Guenther Koehne | 62 | 2027-01-01 | 2026-10-13 |
| CD34+ Transplants for Myeloid Malignancies Busulfan, Melphalan, Fludarabine - Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute | Phase 2 | Not yet recruiting | Guenther Koehne | 50 | 2027-01-01 | 2026-10-13 |
| Arsenic Trioxide to Prevent Relapse in Patients Undergoing Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia or Myelodysplastic Syndrome Allogeneic Hematopoietic Stem Cell Transplantation, Arsenic Trioxide, Biospecimen Collection - Acute Myeloid Leukemia, Myelodysplastic Syndrome | Phase 1 | Not yet recruiting | University of Michigan Rogel Cancer Center | 20 | 2026-11-01 | 2026-07-30 |
| Base-Edited Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of CXCR4-WHIM Busulfan, Palifermin, Plerixafor - WHIM, Warts | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 10 | 2026-10-27 | 2026-10-22 |
| Stem Cell Transplantation for Participants With Germline RUNX1 Associated Blood Cancers Cyclophosphamide, Busulfan, Fludarabine - Core Binding Factor Alpha Subunits, Hematologic Neoplasms | Phase 2 | Not yet recruiting | National Cancer Institute (NCI) | 102 | 2026-10-27 | 2026-10-22 |
| 26DT052; Scaling Busulfan Dose to Body Surface Area in Children Busulfan (BU) - Busulfan Pharmacokinetic Analysis, Busulfan Dosing | Phase 1 | Not yet recruiting | Children's Hospital of Philadelphia | 38 | 2026-09-01 | 2026-08-28 |
| Allogeneic ORCA-Q Stem Cell Transplant for the Treatment of Relapsed and Refractory High Risk Multiple Myeloma Allogeneic Defined Hematopoietic Stem Cells/Immune Cells, Busulfan, Filgrastim - Recurrent Multiple Myeloma, Refractory Multiple Myeloma | Phase 1 | Not yet recruiting | University of California, Davis | 20 | 2026-09-01 | 2026-08-12 |
| A Study of VABu Conditioning Regimen in Allo-HSCT for AML Patients Venetoclax, Azacitidine (AZA), Decitabine 20 mg/m²/day for 5 days - Leukemia, Myeloid, Acute(AML), Hematopoietic Stem Cell Transplantation (HSCT) | Not applicable | Not yet recruiting | The First Affiliated Hospital of Soochow University | 42 | 2026-08-01 | 2026-08-19 |
| Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies Thiotepa, Fludarabine, Busulfan - Acute Lymphocytic Leukemia (ALL), Acute Myeloid Leukemia (AML) | Phase 1 | Recruiting | Sawa Ito, MD | 48 | 2026-07-27 | 2026-08-18 |
| Efficacy and Safety of the CloB2M (Clofarabine Combined With Busulfan and Melphalan) Conditioning Regimen in Allogeneic Hematopoietic Stem Cell Transplantation for Adult Patients With Acute Myeloid Leukemia in First Complete Remission CloB2M (Clofarabine Combined With Busulfan and Melphalan) Conditioning Regimen - AML (Acute Myelogenous Leukemia), Allogeneic Hematopoietic Stem Cell Transplantation Recipient | Not applicable | Not yet recruiting | Institute of Hematology & Blood Diseases Hospital, China | 30 | 2026-07-20 | 2026-06-12 |
| Emapalumab With Post-Transplant Cyclophosphamide, Tacrolimus and Mycophenolate Mofetil for the Prevention of Graft-versus-Host Disease After Donor Reduced-Intensity Hematopoietic Cell Transplant Biospecimen Collection, Busulfan, Computed Tomography - Acute Myeloid Leukemia, Graft Versus Host Disease | Phase 1 | Recruiting | City of Hope Medical Center | 15 | 2026-06-12 | 2026-07-01 |
| TBF Conditioning Regimen for Haploidentical Stem Cell Transplantation in Elderly AML Patients in First Complete Remission Thiotepa, Busulfan (BU), Fludarabine - Acute Myeloid Leukemia | Not applicable | Not yet recruiting | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | 93 | 2026-06-01 | 2026-05-04 |
| Cord Blood Transplantation in Children and Young Adults With Blood Cancer Cord Blood Units, Total Body Irradiation, Cyclophosphamide - Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia | Phase 2 | Recruiting | Memorial Sloan Kettering Cancer Center | 71 | 2026-04-28 | 2026-05-14 |
| The Safety and Efficacy of the Regimen of Thiotepa + Fludarabine +Granulocyte Colony-Stimulating Factor+ Cytarabine + Busulfan in Single Unrelated Umbilical Cord Blood Transplantation Thiotepa, Fludarabine, G-CSF - Hematologic Malignancies | Phase 2 | Not yet recruiting | Institute of Hematology & Blood Diseases Hospital, China | 44 | 2026-02-20 | 2026-03-02 |
| Thiotepa-Containing Conditioning Regimen for Allogeneic HSCT in Chronic Myelomonocytic Leukemia TBF regimen - Chronic Myelomonocytic Leukemia (CMML) | Not applicable | Recruiting | Peking University People's Hospital | 31 | 2026-02-13 | 2026-09-15 |
| Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes Ruxolitinib, Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan - Myelodysplastic/Myeloproliferative Neoplasm, Primary Myelofibrosis | Phase 2 | Recruiting | Fred Hutchinson Cancer Center | 50 | 2026-02-04 | 2026-10-19 |
| Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus Sirolimus, Busulfan, Horse -Anti-thymocyte - Severe Combined Immune Deficiency (SCID) | Phase 1 / Phase 2 | Withdrawn | National Institute of Allergy and Infectious Diseases (NIAID) | 2025-11-26 | 2025-12-01 | |
| Haplo-Cord HSCT for AML/MDS haplo-cord HSCT, Fludarabine, Cytarabine, Busulfan, Antithymocyte Globulin (ATG), Melphalan, Cyclophosphamide (CTX) - MDS (Myelodysplastic Syndrome), AML (Acute Myeloid Leukemia) | Not applicable | Recruiting | Fujian Medical University Union Hospital | 82 | 2025-10-20 | 2026-06-10 |
| Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease Busulfan, Tocilizumab, Eltrombopag - Chronic Granulomatous Disease (CGD) | Phase 1 / Phase 2 | Enrolling by invitation | National Institute of Allergy and Infectious Diseases (NIAID) | 10 | 2025-09-10 | 2026-09-08 |
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Leukemia, Myeloid, Acute (180) · Myelodysplastic Syndromes (152) · Leukemia (148) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (113) · Lymphoma (101) · Lymphoma, Non-Hodgkin (87) · Multiple Myeloma (84) · Hodgkin Disease (75) · Leukemia, Lymphocytic, Chronic, B-Cell (70) · Congenital Abnormalities (65) · Leukemia, Myeloid, Chronic-Phase (60) · Myeloproliferative Disorders (60) · Lymphoma, Large B-Cell, Diffuse (57) · Neoplasms, Plasma Cell (54) · Leukemia, Myeloid, Accelerated Phase (53)
M.D. Anderson Cancer Center (53) · Fred Hutchinson Cancer Center (32) · Masonic Cancer Center, University of Minnesota (30) · Memorial Sloan Kettering Cancer Center (19) · National Cancer Institute (NCI) (15) · National Institute of Allergy and Infectious Diseases (NIAID) (15) · Nanfang Hospital, Southern Medical University (14) · City of Hope Medical Center (12) · St. Jude Children's Research Hospital (12) · University of California, San Francisco (11) · Case Comprehensive Cancer Center (10) · Columbia University (9) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (9) · Children's Oncology Group (7) · Northwestern University (7)
United States (385) · China (49) · France (30) · South Korea (23) · Canada (20) · Italy (20) · United Kingdom (18) · Germany (17) · Belgium (13) · Netherlands (11) · Australia (10) · Austria (10) · Switzerland (10) · Czechia (9) · Israel (9)
Qifa Liu (12) · Elizabeth M Kang, M.D. (7) · Paul Orchard, MD (7) · Richard E. Champlin, MD (5) · Suk S De Ravin, M.D. (5) · Yago Nieto, MD, PHD (5) · Asad Bashey, MD, PhD (4) · Hyoung Jin Kang, M.D., Ph.D (4) · Thomas G. Martin, MD (4) · Uday Popat, MD (4) · Uday R Popat (4) · William I. Bensinger, MD (4) · Barbara Jean Bambach, MD (3) · Boris V. Afanasyev, Professor (3) · Christopher G Kanakry, M.D. (3)
PMID 33951890, PMID 20567002, PMID 21586746, PMID 28380315, PMID 29926156, PMID 30523055, PMID 31553693, PMID 33974366, PMID 34028986, PMID 36184192, PMID 36240296, PMID 36815378, PMID 37267439, PMID 38295280, PMID 38524902, PMID 39796745, PMID 42085603, PMID 10797354, PMID 11468154, PMID 14550808, PMID 16533779, PMID 16572419, PMID 16996130, PMID 17038533, PMID 17889355, PMID 17910637, PMID 18158962, PMID 18162124, PMID 18218852, PMID 18418404, PMID 18550258, PMID 18596741, PMID 18769450, PMID 18997171, PMID 19151774, PMID 19171715, PMID 19179314, PMID 19286996, PMID 19289999, PMID 19304957, PMID 19528536, PMID 19539216, PMID 19633200, PMID 19652199, PMID 19776406, PMID 19826132, PMID 20056794, PMID 20124511, PMID 20308673, PMID 20413658
P30CA015704 (9), P30CA033572 (6), P30CA043703 (6), U10CA031946 (6), P30CA006973 (5), U10CA180886 (5), U10CA098543 (4), U10CA180821 (4), P01CA018029 (3), P01HL036444 (3), P30CA016672 (3), P30CA022453 (3), 2014 (2), P01CA015396 (2), P01CA148600 (2)