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

Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

Not Applicable

Terminated

Conditions: Sickle Cell Disease
Hemoglobin SC

Interventions: Drug: Busulfan
Biological: Campath 1H
Drug: Cyclophosphamide and MESNA

First Posted Date: 2007-12-21

Last Posted Date: 2020-07-31

Lead Sponsor: Tami D. John

Target Recruit Count: 8

Registration Number: NCT00578344

Locations: 🇺🇸
Methodist Hospital, Houston, Texas, United States
🇺🇸
Texas Children's Hospital, Houston, Texas, United States

Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease

Phase 2

Completed

Conditions: Chronic Granulomatous Disease

Interventions: Drug: Busulfan
Biological: Alemtuzumab
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Cyclosporine
Procedure: Stem Cell Infusion

First Posted Date: 2007-12-21

Last Posted Date: 2018-11-09

Lead Sponsor: Baylor College of Medicine

Target Recruit Count: 15

Registration Number: NCT00578643

Locations: 🇺🇸
Texas Children's Hospital, Houston, Texas, United States

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Not Applicable

Terminated

Conditions: Thalassemia

Interventions: Drug: Busulfan
Drug: Fludarabine
Drug: Campath 1H
Drug: Cyclophosphamide
Drug: MESNA

First Posted Date: 2007-12-21

Last Posted Date: 2020-05-01

Lead Sponsor: Baylor College of Medicine

Target Recruit Count: 10

Registration Number: NCT00578292

Locations: 🇺🇸
Texas Children's Hospital, Houston, Texas, United States

Reduced Intensity Preparative Regimen Followed by Stem Cell Transplant (FAB)

Phase 1

Terminated

Conditions: Acute Myelogenous Leukemia
Multiple Myeloma
Lymphoproliferative Disorders
Hematologic Diseases
Myelodysplastic and Myeloproliferative Disorders
Acute Lymphoblastic Leukemia
Chronic Myelogenous Leukemia
Plasma Cell Dyscrasia

Interventions: Drug: Campath
Drug: Busulfan
Drug: Fludarabine
Procedure: Hematopoietic stem cell infusion
Drug: FK-506

First Posted Date: 2007-12-21

Last Posted Date: 2016-05-04

Lead Sponsor: Baylor College of Medicine

Target Recruit Count: 4

Registration Number: NCT00579111

Locations: 🇺🇸
Texas Children's Hospital, Houston, Texas, United States
🇺🇸
The Methodist Hospital, Houston, Texas, United States

Alemtuzumab, Busulfan, and Cyclophosphamide Followed By a Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer

Phase 1

Terminated

Conditions: Graft Versus Host Disease
Leukemia
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Diseases

Interventions: Biological: alemtuzumab
Drug: busulfan
Drug: cyclophosphamide
Drug: methotrexate
Drug: tacrolimus
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: peripheral blood stem cell transplantation

First Posted Date: 2007-11-07

Last Posted Date: 2017-05-24

Lead Sponsor: Fred Hutchinson Cancer Center

Target Recruit Count: 1

Registration Number: NCT00555048

Locations: 🇺🇸
Fred Hutchinson Cancer Research Center, Seattle, Washington, United States
🇺🇸
Seattle Cancer Care Alliance, Seattle, Washington, United States

Donor Stem Cell Transplant in Treating Young Patients With Acute Myeloid Leukemia With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory or Relapsed Acute Myelogenous Leukemia

Phase 2

Completed

Conditions: Leukemia

Interventions: Biological: anti-thymocyte globulin
Drug: busulfan
Drug: cyclophosphamide
Drug: cyclosporine
Drug: methotrexate
Drug: methylprednisolone
Drug: tacrolimus
Other: laboratory biomarker analysis
Other: pharmacological study
Procedure: allogeneic bone marrow transplantation
Procedure: allogeneic hematopoietic stem cell transplantation

First Posted Date: 2007-11-04

Last Posted Date: 2020-04-16

Lead Sponsor: Children's Oncology Group

Target Recruit Count: 158

Registration Number: NCT00553202

Locations: 🇺🇸
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus, Atlanta, Georgia, United States
🇺🇸
Alvin and Lois Lapidus Cancer Institute at Sinai Hospital, Baltimore, Maryland, United States
🇺🇸
Nationwide Children's Hospital, Columbus, Ohio, United States

and more 49 locations

Different Therapies in Treating Infants With Newly Diagnosed Acute Leukemia

Not Applicable

Conditions: Leukemia

First Posted Date: 2007-10-30

Last Posted Date: 2019-07-30

Lead Sponsor: Dutch Childhood Oncology Group

Target Recruit Count: 445

Registration Number: NCT00550992

Locations: 🇨🇿
University Hospital Motol, Prague, Czechia
🇫🇷
CHR Hotel Dieu, Nantes, France
🇺🇸
Children's Hospital and Regional Medical Center - Seattle, Seattle, Washington, United States

and more 10 locations

AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders

Not Applicable

Completed

Conditions: Leukemia
Lymphoma
Myelodysplastic Syndromes

Interventions: Biological: filgrastim
Drug: Cyclophosphamide
Drug: Busulfan
Drug: Cyclosporine
Drug: Fludarabine phosphate
Drug: Mycophenolate Mofetil
Drug: Melphalan
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: umbilical cord blood transplantation
Radiation: Fractionated total body irradiation
Radiation: total-body irradiation

First Posted Date: 2007-10-22

Last Posted Date: 2024-06-14

Lead Sponsor: City of Hope Medical Center

Target Recruit Count: 10

Registration Number: NCT00547196

Locations: 🇺🇸
City of Hope Medical Center, Duarte, California, United States
🇺🇸
Banner Good Samaritan Medical Center, Phoenix, Arizona, United States