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

Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic Cancer

Phase 2

Terminated

Conditions: Myelodysplastic Syndromes
Lymphoma
Leukemia
Myelodysplastic/Myeloproliferative Diseases

Interventions: Biological: alemtuzumab
Biological: graft-versus-tumor induction therapy
Biological: rituximab
Drug: busulfan
Drug: cyclophosphamide
Drug: fludarabine phosphate
Drug: methotrexate
Drug: tacrolimus
Procedure: allogeneic bone marrow transplantation

First Posted Date: 2009-01-08

Last Posted Date: 2013-12-18

Lead Sponsor: Northside Hospital, Inc.

Target Recruit Count: 36

Registration Number: NCT00818961

Locations: 🇺🇸
Blood and Marrow Transplant Group of Georgia, Atlanta, Georgia, United States

Azacitidine After Allo Blood And Marrow Transplantation (BMT) for Chronic Myelogenous Leukemia (CML)

Phase 2

Completed

Conditions: Stem Cell Transplantation
Leukemia

Interventions: Drug: Fludarabine
Drug: Busulfan
Drug: Thymoglobulin
Drug: Azacitidine
Procedure: Stem Cell Transplant

First Posted Date: 2008-12-22

Last Posted Date: 2021-02-11

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 24

Registration Number: NCT00813124

Locations: 🇺🇸
University of Texas MD Anderson Cancer Center, Houston, Texas, United States

Fludarabine, Busulfan, and Antilymphocyte Globulin Followed by Donor Stem Cell Transplant in Treating Older Patients With Hematological Cancer

Phase 2

Completed

Conditions: Chronic Myeloproliferative Disorders
Leukemia
Lymphoma
Multiple Myeloma and Plasma Cell Neoplasm
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms

First Posted Date: 2008-12-11

Last Posted Date: 2011-05-17

Lead Sponsor: Institut Paoli-Calmettes

Target Recruit Count: 82

Registration Number: NCT00806767

Locations: 🇫🇷
Marseille Institute of Cancer - Institut J. Paoli and I. Calmettes, Marseille, France

Fludarabine, Busulfan, Antithymocyte Globulin, and Donor Stem Cell Transplant in Treating Patients With Multiple Myeloma That Has Not Responded to Treatment

Phase 2

Completed

Conditions: Multiple Myeloma and Plasma Cell Neoplasm

First Posted Date: 2008-12-05

Last Posted Date: 2011-05-16

Lead Sponsor: Institut Paoli-Calmettes

Target Recruit Count: 48

Registration Number: NCT00802568

Locations: 🇫🇷
Marseille Institute of Cancer - Institut J. Paoli and I. Calmettes, Marseille, France

Double Cord Blood Transplant for Patients With Malignant and Non-malignant Disorders

Phase 1

Terminated

Conditions: Neuroblastoma
Immunodeficiencies
Anemia
Leukemia
Lymphoma

Interventions: Radiation: Total Body Irradiation
Drug: Alemtuzumab
Drug: Busulfan
Drug: Melphalan
Drug: Fludarabine
Drug: Phenytoin
Drug: Rabbit Antithymocyte Globulin
Drug: Cyclophosphamide
Drug: Horse Antithymocyte Globulin
Drug: Thiotepa

First Posted Date: 2008-12-04

Last Posted Date: 2019-03-27

Lead Sponsor: Columbia University

Target Recruit Count: 1

Registration Number: NCT00801931

Locations: 🇺🇸
Columbia Presbyterian Medical Center, New York, New York, United States

Autologous and Allogeneic Transplant for Relapsed Lymphoma

Phase 1

Completed

Conditions: Hodgkins Disease
Non-Hodgkin's Lymphoma

Interventions: Drug: Fludarabine
Drug: Busulfan
Drug: Anti-Thymocyte Globulin

First Posted Date: 2008-12-04

Last Posted Date: 2019-03-27

Lead Sponsor: Columbia University

Target Recruit Count: 30

Registration Number: NCT00802113

Locations: 🇺🇸
Children's Memorial Hospital in Chicago, Chicago, Illinois, United States
🇺🇸
Columbia University Medical Center, New York, New York, United States
🇺🇸
Hackensack University Medical Center, Hackensack, New Jersey, United States

and more 3 locations

Busulfan and Fludarabine Followed by Post-transplant Cyclophosphamide

Phase 2

Completed

Conditions: Hematologic Diseases
Myeloma
Lymphoma
Leukemia

Interventions: Drug: Busulfan
Drug: Fludarabine
Drug: Cyclophosphamide

First Posted Date: 2008-12-02

Last Posted Date: 2016-09-19

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 56

Registration Number: NCT00800839

Locations: 🇺🇸
University of Texas MD Anderson Cancer Center, Houston, Texas, United States

Safety Study of Bone Marrow Transplant Using Mismatched Tissue Followed by Chemotherapy

Phase 2

Completed

Conditions: Leukemias
MDS
Lymphomas

Interventions: Drug: Busulfan
Drug: Cyclophosphamide
Radiation: Total body irradiation

First Posted Date: 2008-11-24

Last Posted Date: 2019-03-14

Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Target Recruit Count: 107

Registration Number: NCT00796562

Locations: 🇺🇸
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, United States
🇺🇸
Sidney Kimmel Comprehensive Cancer Center, Baltimore, Maryland, United States