Busulfan

Generic Name: Busulfan

Brand Names: Busulfex, Myleran, Busulfan Fresenius Kabi

Drug Type: Small Molecule

Chemical Formula: C_₆H_₁₄O_₆S_₂

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

Clinical Trials

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

Phase 2

Completed

Conditions: Adult Acute Monoblastic Leukemia
Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11
Myelodysplastic Syndrome
Recurrent Adult Acute Myeloid Leukemia
Adult Acute Megakaryoblastic Leukemia
Adult Acute Monocytic Leukemia
Adult Acute Myeloid Leukemia With Maturation
Adult Acute Myeloid Leukemia With t(9;11)(p21.3;q23.3); MLLT3-MLL
Secondary Myelodysplastic Syndrome
Adult Acute Myeloid Leukemia With Minimal Differentiation

Interventions: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Biological: Anti-Thymocyte Globulin
Drug: Azacitidine
Drug: Busulfan
Drug: Fludarabine Phosphate
Other: Laboratory Biomarker Analysis
Drug: Methotrexate
Other: Pharmacological Study
Drug: Tacrolimus

First Posted Date: 2010-07-23

Last Posted Date: 2022-08-04

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 68

Registration Number: NCT01168219

Locations: 🇺🇸
Northwell Health NCORP, Lake Success, New York, United States
🇺🇸
University of Iowa/Holden Comprehensive Cancer Center, Iowa City, Iowa, United States
🇺🇸
Mount Sinai Hospital, New York, New York, United States

and more 14 locations

Reduced-intensity, Related-donor Bone Marrow Transplantation Followed by High-dose Cyclophosphamide for Hematologic Cancers

Phase 2

Terminated

Conditions: Leukemia
Myelodysplastic Syndrome
Lymphoma

Interventions: Drug: Fludarabine
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Drug: Tacrolimus

First Posted Date: 2010-06-02

Last Posted Date: 2018-07-03

Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Target Recruit Count: 15

Registration Number: NCT01135329

Locations: 🇺🇸
The Sydney Kimmel Comprehensive Cancer Center, Baltimore, Maryland, United States

HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies

Phase 2

Completed

Conditions: Multiple Myeloma
Acute Myelogenous Leukemia
Acute Lymphoblastic Leukemia
Myelodysplastic Syndrome

Interventions: Drug: Thiotepa
Radiation: total body irradiation
Drug: Busulfan
Drug: Melphalan
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Clofarabine
Procedure: (CliniMACS) T-cell depleted PBSC Transplant

First Posted Date: 2010-05-07

Last Posted Date: 2022-08-05

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 422

Registration Number: NCT01119066

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States

Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant

Phase 2

Terminated

Conditions: Leukemia
Lymphoma
Lymphoproliferative Disorder
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms

Interventions: Biological: anti-thymocyte globulin
Biological: donor lymphocytes
Biological: filgrastim
Biological: therapeutic allogeneic lymphocytes
Drug: busulfan
Drug: fludarabine phosphate
Drug: methotrexate
Drug: mycophenolate mofetil
Drug: tacrolimus
Other: reduced-intensity transplant conditioning procedure
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: peripheral blood stem cell transplantation

First Posted Date: 2010-05-06

Last Posted Date: 2017-03-24

Lead Sponsor: Alliance for Clinical Trials in Oncology

Target Recruit Count: 6

Registration Number: NCT01118013

Locations: 🇺🇸
Arthur G. James Cancer Hospital and Richard J. Solove Research Institute at Ohio State University Comprehensive Cancer Center, Columbus, Ohio, United States
🇺🇸
Greenebaum Cancer Center at University of Maryland Medical Center, Baltimore, Maryland, United States
🇺🇸
Florida Hospital Cancer Institute at Florida Hospital Orlando, Orlando, Florida, United States

and more 7 locations