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 & Melphalan Conditioning for Autologous Stem Cell Transplant (ASCT) and Lenalidomide Maintenance

Phase 2

Completed

Conditions: Multiple Myeloma

Interventions: Drug: Busulfan
Drug: Melphalan
Drug: Lenalidomide

First Posted Date: 2012-10-08

Last Posted Date: 2022-09-21

Lead Sponsor: University Health Network, Toronto

Target Recruit Count: 78

Registration Number: NCT01702831

Locations: 🇨🇦
Saint John Regional Hospital, 5DN Research Department, 400 University Ave, Saint John, New Brunswick, Canada
🇨🇦
Cross Cancer Institute 11560 University Ave, Edmonton, Alberta, Canada
🇨🇦
Queen Elizabeth II Health Sciences Centre., Halifax, Nova Scotia, Canada

and more 7 locations

Gemcitabine Hydrochloride, Clofarabine, and Busulfan Before Donor Stem Cell Transplant in Treating Patients With Refractory B-Cell or T-Cell Non-Hodgkin Lymphoma or Hodgkin Lymphoma

Phase 1

Completed

Conditions: Refractory Hodgkin Lymphoma
Refractory B-Cell Non-Hodgkin Lymphoma
Refractory T-Cell Non-Hodgkin Lymphoma
Hematopoietic Cell Transplantation Recipient

Interventions: Procedure: Allogeneic Bone Marrow Transplantation
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Biological: Anti-Thymocyte Globulin
Drug: Busulfan
Drug: Clofarabine
Drug: Gemcitabine Hydrochloride
Drug: Mycophenolate Mofetil
Procedure: Peripheral Blood Stem Cell Transplantation
Other: Pharmacological Study
Biological: Rituximab
Drug: Tacrolimus

First Posted Date: 2012-10-05

Last Posted Date: 2024-06-07

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 64

Registration Number: NCT01701986

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

Busulfan and Cyclophosphamide Followed By ALLO BMT

Not Applicable

Terminated

Conditions: Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia
Acute Myeloid Leukemia

Interventions: Drug: Allopurinol
Drug: Keppra
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Biological: Allogeneic hematopoietic stem cell transplant
Biological: Filgrastim
Biological: antithymocyte globulin

First Posted Date: 2012-09-14

Last Posted Date: 2021-04-13

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 5

Registration Number: NCT01685411

Locations: 🇺🇸
Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

Not Applicable

Recruiting

Conditions: Immunodeficiencies
Hemoglobinopathies
Hematologic Disorders

Interventions: Drug: Busulfan
Drug: Fludarabine
Radiation: Total body irradiation
Biological: Stem cell transplant
Drug: Keppra

First Posted Date: 2012-08-16

Last Posted Date: 2024-07-19

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 30

Registration Number: NCT01666080

Locations: 🇺🇸
Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Not Applicable

Recruiting

Conditions: Omenn's Syndrome
Wiskott-Aldrich Syndrome
Bare Lymphocyte Syndrome
SCID
Hemophagocytic Lymphohistiocytosis
Langerhan's Cell Histiocytosis
Reticular Dysgenesis
Common Variable Immunodeficiency
Hyper IgM Syndrome
Griscelli Syndrome

Interventions: Drug: Alemtuzumab 0.3 mg
Drug: Cyclophosphamide
Drug: Melphalan
Drug: Fludarabine phosphate 40 mg
Drug: Alemtuzumab 0.2 mg
Drug: Busulfan
Drug: Fludarabine phosphate 30 mg
Biological: Stem Cell Transplantation
Drug: MESNA

First Posted Date: 2012-07-27

Last Posted Date: 2024-01-26

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 30

Registration Number: NCT01652092

Locations: 🇺🇸
Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States

Busulfan/Clofarabine + Allogeneic Stem Cell Transplantation

Phase 2

Completed

Conditions: Myelodysplastic Syndrome
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia

Interventions: Drug: Busulfan
Drug: Clofarabine
Procedure: Allogeneic Stem Cell Infusion

First Posted Date: 2012-07-18

Last Posted Date: 2017-07-13

Lead Sponsor: Massachusetts General Hospital

Target Recruit Count: 34

Registration Number: NCT01643668

Locations: 🇺🇸
Dana-Farber Cancer Institute, Boston, Massachusetts, United States
🇺🇸
Brigham and Women's Hospital, Boston, Massachusetts, United States
🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States