Busulfan
- Generic Name
- Busulfan
- Brand Names
- Busulfex, Myleran, Busulfan Fresenius Kabi
- Drug Type
- Small Molecule
- Chemical Formula
- C6H14O6S2
- CAS Number
- 55-98-1
- Unique Ingredient Identifier
- G1LN9045DK
- Background
Busulfan is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow. It is not a structural analog of the nitrogen mustards. It has been used in the palliative treatment of chronic myeloid leukemia (myeloid leukemia, chronic), but although symptomatic relief is provided, no permanent remission is brought about. According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), busulfan is listed as a known carcinogen.
- Indication
For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous (myeloid, myelocytic, granulocytic) leukemia (FDA has designated busulfan as an orphan drug for this use). It is also used as a component of pretransplant conditioning regimens in patients undergoing bone marrow transplantation for acute myeloid leukemia and nonmalignant diseases.
- Associated Conditions
- Chronic Myelogenous Leukemia (CML), Essential Thrombocythemia (ET), Polycythemia Vera (PV)
- Associated Therapies
- Conditioning regimens for allogeneic stem cell transplantation therapy
The Use of N-acetylcysteine for Thrombotic Events After Allogenic Hematopoietic Stem Cell Transplantation
- Conditions
- Thrombotic Disorder
- Interventions
- First Posted Date
- 2023-06-18
- Last Posted Date
- 2023-06-18
- Lead Sponsor
- The First Affiliated Hospital of Soochow University
- Target Recruit Count
- 260
- Registration Number
- NCT05907486
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
- Conditions
- Hematologic MalignancyMinimal Residual DiseasePTPN11 Gene MutationMonosomy 7Chromosome AbnormalityAcute LeukemiaRemissionTP53Intrachromosomal Amplification of Chromosome 21Myelodysplasia
- Interventions
- First Posted Date
- 2023-02-21
- Last Posted Date
- 2024-10-21
- Lead Sponsor
- Masonic Cancer Center, University of Minnesota
- Target Recruit Count
- 150
- Registration Number
- NCT05735717
- Locations
- 🇺🇸
University of Minnesota Masonic Cancer Center, Minneapolis, Minnesota, United States
CD34+ Enriched Transplants to Treat Myelodysplastic Syndrome
- Conditions
- Graft Vs Host DiseaseGraft-versus-host-diseaseMyelodysplastic Syndromes
- Interventions
- Device: CliniMACS CD34+ enriched, T-cell depleted peripheral blood stem cell (PBSC)
- First Posted Date
- 2022-11-15
- Last Posted Date
- 2025-04-15
- Lead Sponsor
- Guenther Koehne
- Target Recruit Count
- 50
- Registration Number
- NCT05617625
- Locations
- 🇺🇸
Miami Cancer Institute, Miami, Florida, United States
A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies
- Conditions
- Myeloid MalignancyHematologic Malignancy
- Interventions
- Drug: Anti-thymocyte globulin (ATG)Radiation: Total Body Irradiation (radiation treatment)Drug: Bone marrow infusion
- First Posted Date
- 2022-10-14
- Last Posted Date
- 2024-07-10
- Lead Sponsor
- St. Jude Children's Research Hospital
- Target Recruit Count
- 3
- Registration Number
- NCT05579769
- Locations
- 🇺🇸
St. Jude Children's Research Hospital, Memphis, Tennessee, United States
Uproleselan With Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for AML
- Conditions
- Acute Myeloid LeukemiaPediatric Cancer
- Interventions
- First Posted Date
- 2022-10-06
- Last Posted Date
- 2024-08-19
- Lead Sponsor
- Malika Kapadia
- Target Recruit Count
- 1
- Registration Number
- NCT05569512
- Locations
- 🇺🇸
Helen DeVos Children's Hospital/Spectrum Health, Grand Rapids, Michigan, United States
🇺🇸Memorial Sloan Kettering Cancer Center, New York, New York, United States
🇺🇸Dana-Farber Cancer Institute, Boston, Massachusetts, United States
CD34+ Transplants for Leukemia and Lymphoma
- Conditions
- Leukemia, Lymphocytic, AcuteLeukemia, Myeloid, Acute
- Interventions
- Radiation: Total Body Irradiation (TBI)
- First Posted Date
- 2022-10-04
- Last Posted Date
- 2025-04-15
- Lead Sponsor
- Guenther Koehne
- Target Recruit Count
- 100
- Registration Number
- NCT05565105
- Locations
- 🇺🇸
Baptist Health South Florida/Miami Cancer Institute, Miami, Florida, United States
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
- Conditions
- Myelodysplastic SyndromeAcute Myeloid Leukemia
- Interventions
- Procedure: Biospecimen CollectionOther: Best PracticeProcedure: Allogeneic Hematopoietic Stem Cell TransplantationProcedure: Bone Marrow AspirationProcedure: Biopsy ProcedureProcedure: Mutation Carrier ScreeningProcedure: Bone Marrow BiopsyProcedure: Echocardiography TestProcedure: Multigated Acquisition ScanDrug: Liposome-encapsulated Daunorubicin-CytarabineProcedure: Chest RadiographyProcedure: Computed TomographyProcedure: Positron Emission TomographyDrug: Placebo AdministrationRadiation: Total-Body Irradiation
- First Posted Date
- 2022-10-03
- Last Posted Date
- 2025-05-21
- Lead Sponsor
- National Cancer Institute (NCI)
- Target Recruit Count
- 2000
- Registration Number
- NCT05564390
- Locations
- 🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸Mercy Hospital South, Saint Louis, Missouri, United States
🇺🇸Siteman Cancer Center-South County, Saint Louis, Missouri, United States
ChiCGB vs BEAM in High-risk or R/R Lymphomas
- Conditions
- Lymphoma, Large B-Cell, DiffuseLymphoma, T-Cell
- Interventions
- Procedure: Autologous hematopoietic stem cell transplant
- First Posted Date
- 2022-07-20
- Last Posted Date
- 2022-07-25
- Lead Sponsor
- Sichuan University
- Target Recruit Count
- 306
- Registration Number
- NCT05466318
- Locations
- 🇨🇳
Chengdu Third People's Hospital, Chendu, Sichuan, China
🇨🇳People's Hospital of Deyang City, Deyang, Deyang, China
🇨🇳West China Hospital, Sichuan University, Chendu, Sichuan, China
Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
- Conditions
- Chronic Granulomatous Disease
- Interventions
- Biological: Pheripheral blood stem cellsDrug: TociluzumabDrug: Emapalumab-IzsgDrug: Total Body Irradiation
- First Posted Date
- 2022-07-18
- Last Posted Date
- 2025-05-20
- Target Recruit Count
- 50
- Registration Number
- NCT05463133
- Locations
- 🇺🇸
National Institutes of Health Clinical Center, Bethesda, Maryland, United States
Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome
- Conditions
- Mixed Phenotype Acute LeukemiaAcute Myeloid LeukemiaMyelodysplastic SyndromeAcute Lymphoblastic Leukemia
- Interventions
- Procedure: Biospecimen CollectionProcedure: Bone Marrow AspirationProcedure: Echocardiography TestProcedure: Haploidentical Hematopoietic Cell TransplantationProcedure: Lumbar PunctureProcedure: Matched Unrelated Donor Hematopoietic Cell TransplantationProcedure: Multigated Acquisition ScanProcedure: Myeloablative ConditioningOther: Quality-of-Life AssessmentOther: Questionnaire AdministrationRadiation: Total-Body IrradiationProcedure: T-Cell Depletion Therapy
- First Posted Date
- 2022-07-14
- Last Posted Date
- 2025-05-08
- Lead Sponsor
- Children's Oncology Group
- Target Recruit Count
- 435
- Registration Number
- NCT05457556
- Locations
- 🇺🇸
Children's Hospital of Alabama, Birmingham, Alabama, United States
🇺🇸C S Mott Children's Hospital, Ann Arbor, Michigan, United States
🇺🇸Children's Hospital of Michigan, Detroit, Michigan, United States
Drug Development Updates
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