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

T-Cell Depleted Double UCB for Refractory AML

Phase 2

Terminated

Conditions: Acute Myelogenous Leukemia
Refractory Acute Myelogenous Leukemia

Interventions: Drug: Allopurinol
Drug: Fludarabine
Radiation: Total body irradiation
Drug: Cyclophosphamide
Drug: Levetiracetam
Drug: Busulfan
Biological: Umbilical Cord Blood Transplantation
Biological: Interleukin-2

First Posted Date: 2011-11-03

Last Posted Date: 2017-12-28

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 3

Registration Number: NCT01464359

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

Cyclophosphamide for Prevention of Graft-Versus-Host Disease After Allogeneic Peripheral Blood Stem Cell Transplantation in Patients With Hematological Malignancies

Phase 2

Completed

Conditions: Accelerated Phase Chronic Myelogenous Leukemia
Adult Acute Lymphoblastic Leukemia in Remission
Adult Acute Megakaryoblastic Leukemia (M7)
Adult Acute Myeloid Leukemia in Remission
Adult Erythroleukemia (M6a)
Adult Nasal Type Extranodal NK/T-cell Lymphoma
Adult Pure Erythroid Leukemia (M6b)
Anaplastic Large Cell Lymphoma
Angioimmunoblastic T-cell Lymphoma
Blastic Phase Chronic Myelogenous Leukemia

Interventions: Drug: cyclophosphamide
Drug: cyclosporine
Procedure: peripheral blood stem cell transplantation
Radiation: total-body irradiation
Drug: fludarabine phosphate
Drug: busulfan
Procedure: allogeneic hematopoietic stem cell transplantation

First Posted Date: 2011-09-02

Last Posted Date: 2017-05-19

Lead Sponsor: Fred Hutchinson Cancer Center

Target Recruit Count: 43

Registration Number: NCT01427881

Locations: 🇺🇸
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium, Seattle, Washington, United States

Vorinostat With Gemcitabine, Busulfan, and Melphalan With Stem Cell Transplant (SCT) in Relapsed or Refractory Lymphoid Malignancies

Phase 1

Completed

Conditions: Lymphoma

Interventions: Drug: Vorinostat
Drug: Gemcitabine
Drug: Busulfan
Drug: Melphalan
Procedure: Stem Cell Infusion
Drug: Rituximab
Drug: G-CSF
Drug: Palifermin
Drug: Dexamethasone acetate

First Posted Date: 2011-08-22

Last Posted Date: 2015-11-18

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 78

Registration Number: NCT01421173

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

A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma

Phase 3

Completed

Conditions: Myeloma

Interventions: Drug: Busulfan
Drug: Melphalan
Other: Questionnaire
Drug: High Dose Melphalan
Drug: G-CSF
Procedure: Stem cell transplant

First Posted Date: 2011-08-10

Last Posted Date: 2020-04-21

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 205

Registration Number: NCT01413178

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

Alloreactive Haploidentical Natural Killer (NK) Cells With Busulfan and Fludarabine/ATG

Phase 2

Completed

Conditions: Chronic Myelogenous Leukemia
Leukemia

Interventions: Drug: Fludarabine
Drug: Busulfan
Drug: Interleukin-2
Drug: Anti-Thymocyte Globulin
Procedure: Allogeneic related Stem Cell Transplant
Drug: Tacrolimus
Drug: Methotrexate
Drug: G-CSF

First Posted Date: 2011-07-11

Last Posted Date: 2016-02-03

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 6

Registration Number: NCT01390402

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

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

Phase 2

Completed

Conditions: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Childhood Acute Lymphoblastic Leukemia in Remission
Adult Acute Lymphoblastic Leukemia in Remission
Adult Acute Myeloid Leukemia in Remission
Childhood Myelodysplastic Syndromes
Chronic Eosinophilic Leukemia
Chronic Myelomonocytic Leukemia
Chronic Neutrophilic Leukemia
Essential Thrombocythemia
Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue

Interventions: Drug: Fludarabine
Drug: Busulfan
Radiation: Total Body Irradiation (TBI)
Biological: Donor Lymphocyte Infusion (DLI)
Drug: Cyclophosphamide (CY)
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Device: Allogeneic hematopoietic stem cell transplantation
Procedure: Peripheral blood stem cell transplantation (PBSCT)

First Posted Date: 2011-06-29

Last Posted Date: 2022-11-17

Lead Sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University

Target Recruit Count: 40

Registration Number: NCT01384513

Locations: 🇺🇸
Thomas Jefferson University, Philadelphia, Pennsylvania, United States

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Phase 1

Completed

Conditions: Adenosine Deaminase Deficiency
Severe Combined Immunodeficiencies (SCID)

Interventions: Other: Haematopoietic Stem Cell Transplantation (HSCT)
Genetic: Infusion of autologous EFS-ADA LV CD34+ cells
Drug: Busulfan
Drug: Peg-Ada

First Posted Date: 2011-06-27

Last Posted Date: 2021-09-16

Lead Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust

Target Recruit Count: 36

Registration Number: NCT01380990

Locations: 🇬🇧
Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

Cord Blood Transplantation for Patients With Cancer

Phase 2

Terminated

Conditions: Hematological Disease

Interventions: Drug: Melphalan
Drug: Antithymocyte Globulin (ATG)
Drug: Fludarabine
Drug: Busulfan
Drug: Total Body Irradiation (TBI)

First Posted Date: 2011-05-24

Last Posted Date: 2015-04-30

Lead Sponsor: University of Chicago

Target Recruit Count: 1

Registration Number: NCT01359254

Locations: 🇺🇸
University of Chicago, Chicago, Illinois, United States

A Research Study of Bone Marrow Transplantation From Unrelated or Partially Matched Related Donors

Phase 2

Completed

Conditions: Hematological Malignancies

Interventions: Radiation: Total Body Irradiation
Drug: Cyclophosphamide
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Drug: Fludarabine
Drug: Busulfan
Genetic: Hematopoietic stem cell transplantation

First Posted Date: 2011-05-06

Last Posted Date: 2022-12-21

Lead Sponsor: Sidney Kimmel Cancer Center at Thomas Jefferson University

Target Recruit Count: 80

Registration Number: NCT01349101

Locations: 🇺🇸
Thomas Jefferson University, Philadelphia, Pennsylvania, United States

Pilot Study of Leuprolide to Improve Immune Function After Allogeneic Bone Marrow Transplantation

Phase 2

Completed

Conditions: Myelodysplastic Syndrome
Acute Lymphocytic Leukemia
Acute Myelogenous Leukemia
Chronic Myelogenous Leukemia
Chronic Myelomonocytic Leukemia

Interventions: Procedure: First Allogeneic Bone Marrow Transplant (BMT)
Drug: Leuprolide
Drug: 18F FLT
Drug: Cyclophosphamide
Drug: Methotrexate
Drug: Busulfan
Drug: Tacrolimus
Drug: Fludarabine
Radiation: Total Body Irradiation
Procedure: Second Allogeneic Bone Marrow Transplantation

First Posted Date: 2011-04-20

Last Posted Date: 2021-03-18

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 76

Registration Number: NCT01338987

Locations: 🇺🇸
Childrens National Medical Center, Washington, District of Columbia, United States
🇺🇸
National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States
🇺🇸
University of Oklahoma, Oklahoma City, Oklahoma, United States

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