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

High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease

Phase 1

Active, not recruiting

Conditions: Chronic Granulomatous Disease Transplant

Interventions: Drug: Alemtuzumab
Drug: Busulfan
Drug: Sirolimus
Drug: Cyclophosphamide
Radiation: Total Body Irradiation
Biological: Peripheral blood stem cells

First Posted Date: 2015-12-14

Last Posted Date: 2024-12-10

Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Target Recruit Count: 45

Registration Number: NCT02629120

Locations: 🇺🇸
National Institutes of Health Clinical Center, Bethesda, Maryland, United States

Trial of GVHD Prophylasxis With PTCy or Thymoglobulin in Unrelated SCT

Phase 2

Terminated

Conditions: Myelodysplastic Syndromes
Leukemia, Chronic Myeloid
Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative

Interventions: Procedure: Unrelated allogeneic stem cell transplantation
Drug: Busulfan
Drug: Fludarabine monophosphate
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Drug: Thymoglobulin
Drug: Cyclophosphamide

First Posted Date: 2015-12-11

Last Posted Date: 2019-04-05

Lead Sponsor: St. Petersburg State Pavlov Medical University

Target Recruit Count: 32

Registration Number: NCT02627573

Locations: 🇷🇺
First Pavlov State Medical University of St. Petersburg, Saint-Petersburg, Russian Federation

Chemo Sensitization Before Hematopoietic Stem Cell Transplantation in Patients With Acute Leukemia in Complete Remission

Phase 2

Conditions: Acute Lymphoid Leukemia
Acute Myeloid Leukemia

Interventions: Drug: Busulfan
Drug: Cyclophosphamide
Drug: CXCR4 Antagonist
Procedure: Hematopoietic Stem Cell Transplantation

First Posted Date: 2015-11-16

Last Posted Date: 2020-01-14

Lead Sponsor: Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran

Target Recruit Count: 20

Registration Number: NCT02605460

Locations: 🇲🇽
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran, Mexico City, Distrito Federal, Mexico

Lenalidomide Combined With Vorinostat/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Diffuse Large B-Cell Lymphoma of the ABC Subtype

Phase 1

Terminated

Conditions: Lymphoma

Interventions: Drug: Lenalidomide
Drug: Vorinostat
Drug: Gemcitabine
Drug: Busulfan
Drug: Melphalan
Drug: Rituximab
Drug: Dexamethasone
Drug: Caphosol
Drug: Glutamine
Drug: Pyridoxine
Drug: Enoxaparin
Procedure: Stem Cell Transplant
Drug: Palifermin

First Posted Date: 2015-10-28

Last Posted Date: 2019-12-13

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 8

Registration Number: NCT02589145

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

MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

Phase 2

Completed

Conditions: Epidermolysis Bullosa

Interventions: Drug: Thymoglobulin
Drug: Cyclophosphamide
Drug: Fludarabine
Radiation: Total Body Irradiation
Procedure: Bone marrow infusion
Drug: Tacrolimus
Drug: Mycophenolate Mofetil
Biological: Donor mesenchymal stem cell infusions
Drug: Busulfan

First Posted Date: 2015-10-21

Last Posted Date: 2023-08-01

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 17

Registration Number: NCT02582775

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

Testing a Reduced Conditioning Regimen FB2A2 Preceded by a Fractionated Radio-immunotherapy (RIT) With 90Y-Epratuzumab Before Allogeneic Stem Cell Transplantation for Patients With Lymphocyte B CD22 Positive Acute Lymphoblastic Leukemia

Phase 1

Withdrawn

Conditions: Lymphocyte B CD22 Positive Acute Lymphoblastic Leukemia

First Posted Date: 2015-10-16

Last Posted Date: 2017-01-19

Lead Sponsor: Nantes University Hospital

Registration Number: NCT02577094

VP-16, Ifosfamide, Dexamethasone, L-asparaginase Chemotherapy in Patients With Extranodal Natural Killer T Cell Lymphoma (VIDL+ASCT)

Phase 2

Conditions: Extranodal NK-T-Cell Lymphoma

Interventions: Drug: Etoposide
Drug: Ifosfamide
Drug: Dexamethasone
Drug: L-asparaginase
Drug: Busulfan
Drug: Melphalan

First Posted Date: 2015-09-09

Last Posted Date: 2020-10-22

Lead Sponsor: Samsung Medical Center

Target Recruit Count: 27

Registration Number: NCT02544425

Locations: 🇰🇷
Samsung Medical Center, Seoul, Korea, Republic of

Romidepsin Maintenance After Allogeneic Stem Cell Transplantation

Phase 1

Active, not recruiting

Conditions: Cutaneous T-cell Lymphoma
T-Prolymphocytic Leukemia
T-Large Granulocytic Leukemia
T-Lymphoblastic Leukemia/Lymphoma
Peripheral T-Cell Lymphoma

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

First Posted Date: 2015-07-31

Last Posted Date: 2024-10-29

Lead Sponsor: Ohio State University Comprehensive Cancer Center

Target Recruit Count: 23

Registration Number: NCT02512497

Locations: 🇺🇸
The Ohio State University Cancer Center, Columbus, Ohio, United States

Panobinostat, Gemcitabine Hydrochloride, Busulfan, and Melphalan Before Stem Cell Transplant in Treating Patients With Refractory or Relapsed Multiple Myeloma

Phase 2

Completed

Conditions: Recurrent Plasma Cell Myeloma
Plasma Cell Leukemia
Plasmacytoma
Refractory Plasma Cell Myeloma

Interventions: Procedure: Autologous Hematopoietic Stem Cell Transplantation
Drug: Busulfan
Drug: Gemcitabine Hydrochloride
Other: Laboratory Biomarker Analysis
Drug: Melphalan
Drug: Panobinostat
Procedure: Peripheral Blood Stem Cell Transplantation
Other: Pharmacological Study