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Busulfan

Generic Name
Busulfan
Brand Names
Busulfex, Myleran, Busulfan Fresenius Kabi
Drug Type
Small Molecule
Chemical Formula
C6H14O6S2
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

Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome, Acute Myeloid Leukemia, or Myeloproliferative Disorder

Phase 1
Completed
Conditions
Chronic Myeloproliferative Disorders
Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
First Posted Date
2003-01-27
Last Posted Date
2010-09-16
Lead Sponsor
Fred Hutchinson Cancer Center
Target Recruit Count
30
Registration Number
NCT00049634
Locations
🇺🇸

Fred Hutchinson Cancer Research Center, Seattle, Washington, United States

🇺🇸

Seattle Cancer Care Alliance, Seattle, Washington, United States

Combination Chemotherapy With or Without Monoclonal Antibody Therapy in Treating Patients With AML Leukemia

Phase 3
Completed
Conditions
Leukemia
Interventions
Biological: sargramostim
Procedure: Allogeneic HCT
Drug: gemtuzumab ozogamicin (GO)
Procedure: Autologous HCT
First Posted Date
2003-01-27
Last Posted Date
2023-06-15
Lead Sponsor
Eastern Cooperative Oncology Group
Target Recruit Count
657
Registration Number
NCT00049517
Locations
🇺🇸

Community Medical Center, Missoula, Montana, United States

🇺🇸

Penn State Cancer Institute at Milton S. Hershey Medical Center, Hershey, Pennsylvania, United States

🇺🇸

Mercy and Unity Cancer Center at Mercy Hospital, Coon Rapids, Minnesota, United States

and more 96 locations

Busulfan and Cyclophosphamide Followed by Bone Marrow Transplantation in Treating Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome

Phase 2
Completed
Conditions
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases
Leukemia
First Posted Date
2003-01-27
Last Posted Date
2012-06-12
Lead Sponsor
Northwestern University
Registration Number
NCT00004896
Locations
🇺🇸

Robert H. Lurie Comprehensive Cancer Center at Northwestern University, Chicago, Illinois, United States

Busulfan and Etoposide Followed by Peripheral Blood Stem Cell Transplant and Low-Dose Aldesleukin in Treating Patients With Acute Myeloid Leukemia

Phase 2
Completed
Conditions
Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
Recurrent Childhood Acute Myeloid Leukemia
Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
Recurrent Adult Acute Myeloid Leukemia
Adult Acute Myeloid Leukemia in Remission
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Adult Acute Myeloid Leukemia With Del(5q)
Childhood Acute Myeloid Leukemia in Remission
Interventions
Biological: aldesleukin
Procedure: peripheral blood stem cell transplantation
First Posted Date
2003-01-27
Last Posted Date
2017-06-05
Lead Sponsor
Fred Hutchinson Cancer Center
Target Recruit Count
30
Registration Number
NCT00003875
Locations
🇺🇸

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium, Seattle, Washington, United States

Bone Marrow Transplant in Treating Patients With Hematologic Cancers

Phase 2
Completed
Conditions
Multiple Myeloma and Malignant Plasma Cell Neoplasms
Myelodysplastic/Myeloproliferative Neoplasms
Leukemia
Chronic Myeloproliferative Disorders
Myelodysplastic Syndromes
Interventions
Radiation: Fractionated Total Body Irradiation (FTBI)
First Posted Date
2003-01-27
Last Posted Date
2020-12-10
Lead Sponsor
H. Lee Moffitt Cancer Center and Research Institute
Target Recruit Count
125
Registration Number
NCT00005797
Locations
🇺🇸

H. Lee Moffitt Cancer Center and Research Institute at University of South Florida, Tampa, Florida, United States

Study of Gelonin Purging of Autologous Stem Cells for Transplantation

Phase 1
Terminated
Conditions
Myelodysplastic Syndrome
Acute Myelogenous Leukemia
Interventions
First Posted Date
2002-08-15
Last Posted Date
2018-10-31
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
3
Registration Number
NCT00043810
Locations
🇺🇸

UT MD Anderson Cancer Center, Houston, Texas, United States

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

Phase 2
Terminated
Conditions
Sickle Cell Anemia
Hemoglobinopathy
Thalassemia
First Posted Date
2002-06-28
Last Posted Date
2020-01-18
Lead Sponsor
Baylor College of Medicine
Target Recruit Count
15
Registration Number
NCT00040469
Locations
🇺🇸

Texas Children's Hospital, Houston, Texas, United States

🇺🇸

The Methodist Hospital, Houston, Texas, United States

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

Phase 2
Terminated
Conditions
Hemoglobinopathies
Anemia, Sickle Cell
Hemoglobin SC Disease
Thalassemia
Thalassemia Major
Interventions
First Posted Date
2002-05-01
Last Posted Date
2013-05-01
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Target Recruit Count
2
Registration Number
NCT00034528
Locations
🇺🇸

Dana-Farber Cancer Institute/Harvard Cancer Center, Brigham and Women's Hospital and Massachusetts General Hospital, Boston, Massachusetts, United States

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

Phase 2
Completed
Conditions
Hematologic Diseases
Beta-Thalassemia
Hematopoietic Stem Cell Transplantation
Anemia, Sickle Cell
First Posted Date
2002-01-11
Last Posted Date
2016-07-29
Lead Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Target Recruit Count
30
Registration Number
NCT00029380
Locations
🇺🇸

Medical University of South Carolina, Charleston, South Carolina, United States

🇺🇸

Children's Hospital, Oakland, Oakland, California, United States

🇺🇸

Children's National Medical Center, Washington, District of Columbia, United States

and more 12 locations

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

Not Applicable
Terminated
Conditions
Immunologic Deficiency Syndromes
Chediak-Higashi Syndrome
Common Variable Immunodeficiency
Graft Versus Host Disease
X-Linked Lymphoproliferative Syndrome
Familial Erythrophagocytic Lymphohistiocytosis
Hemophagocytic Lymphohistiocytosis
X-linked Agammaglobulinemia
Wiskott-Aldrich Syndrome
Chronic Granulomatous Disease
First Posted Date
2000-07-06
Last Posted Date
2009-10-15
Lead Sponsor
Fairview University Medical Center
Registration Number
NCT00006054
Locations
🇺🇸

Fairview University Medical Center, Minneapolis, Minnesota, United States

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