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, and Bortezomib Before First-Line Stem Cell Transplant in Treating Patients With Multiple Myeloma

Phase 2

Completed

Conditions: DS Stage II Plasma Cell Myeloma
Refractory Plasma Cell Myeloma
DS Stage I Plasma Cell Myeloma
DS Stage III Plasma Cell Myeloma

Interventions: Drug: Busulfan
Drug: Melphalan
Drug: Bortezomib
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Procedure: Peripheral Blood Stem Cell Transplantation

First Posted Date: 2012-05-24

Last Posted Date: 2020-08-20

Lead Sponsor: Albert Einstein College of Medicine

Target Recruit Count: 19

Registration Number: NCT01605032

Locations: 🇺🇸
Rutgers Cancer Institute of New Jersey, New Brunswick, New Jersey, United States
🇺🇸
NYU Cancer Institute, New York, New York, United States
🇺🇸
Albert Einstein College of Medicine, Bronx, New York, United States

and more 1 locations

Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation

Phase 2

Terminated

Conditions: Myeloid Malignancy
Bone Marrow Failure Syndrome
Severe Immune Dysregulation
Metabolic Disease
Transfusion-dependent Red Blood Cell (RBC) Defect
Congenital Immunodeficiency

Interventions: Drug: Busulfan
Drug: Alemtuzumab
Drug: Fludarabine
Drug: Clofarabine

First Posted Date: 2012-05-11

Last Posted Date: 2020-02-26

Lead Sponsor: University of California, San Francisco

Target Recruit Count: 16

Registration Number: NCT01596699

Locations: 🇺🇸
University of California, San Francisco, San Francisco, California, United States

Allogeneic Transplantation Using Timed Sequential Busulfan and Fludarabine Conditioning

Phase 2

Completed

Conditions: Acute Myeloid Leukemia
Non-Hodgkins Lymphoma
Multiple Myeloma
Myelodysplastic Syndrome
Leukemia
Myeloproliferative Diseases
Acute Lymphocytic Leukemia
Chronic Myeloid Leukemia
Chronic Lymphocytic Leukemia
Hodgkins Lymphoma

Interventions: Drug: Fludarabine monophosphate
Drug: Busulfan
Procedure: Stem Cell Infusion
Drug: Tacrolimus
Drug: Methotrexate
Drug: G-CSF

First Posted Date: 2012-04-06

Last Posted Date: 2023-06-29

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 201

Registration Number: NCT01572662

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

High Dose Busulfan and Bortezomib in Treating Patients With High Risk Multiple Myeloma Undergoing Stem Cell Transplant

Phase 2

Terminated

Conditions: Stage I Multiple Myeloma
Stage III Multiple Myeloma
Refractory Multiple Myeloma
Stage II Multiple Myeloma

Interventions: Other: pharmacological study
Drug: tacrolimus
Drug: sirolimus
Biological: anti-thymocyte globulin
Drug: fludarabine phosphate
Drug: busulfan
Drug: bortezomib
Procedure: allogeneic hematopoietic stem cell transplantation
Other: laboratory biomarker analysis

First Posted Date: 2012-02-16

Last Posted Date: 2017-04-05

Lead Sponsor: Barbara Ann Karmanos Cancer Institute

Target Recruit Count: 1

Registration Number: NCT01534143

Locations: 🇺🇸
Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, United States

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

Phase 1

Suspended

Conditions: Severe Combined Immunodeficiency Disease, X-linked

Interventions: Genetic: CL20-i4-EF1α-hγc-OPT
Drug: Busulfan
Device: CliniMacs

First Posted Date: 2012-01-19

Last Posted Date: 2024-04-17

Lead Sponsor: St. Jude Children's Research Hospital

Target Recruit Count: 28

Registration Number: NCT01512888

Locations: 🇺🇸
University of California-San Francisco, San Francisco, California, United States
🇺🇸
St. Jude Children's Research Hospital, Memphis, Tennessee, United States
🇺🇸
Seattle Children's Research Institute, Seattle, Washington, United States