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

PTCy and Ruxolitinib GVHD Prophylaxis in Myelofibrosis

Phase 1

Completed

Conditions: Primary Myelofibrosis
Myeloproliferative Disorders

Interventions: Procedure: Allogeneic hematopoietic stem cell transplantation
Drug: Busulfan
Drug: Fludarabine monophosphate
Drug: Cyclophosphamide
Drug: Ruxolitinib

First Posted Date: 2016-06-20

Last Posted Date: 2019-04-04

Lead Sponsor: St. Petersburg State Pavlov Medical University

Target Recruit Count: 20

Registration Number: NCT02806375

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

GVHD Prophylaxis With Post-transplantation Bendamustine in Refractory Leukemia

Phase 1

Completed

Conditions: Mixed-Lineage Acute Leukemias
Leukemia, Acute Lymphoblastic
Acute Myeloid Leukemia

Interventions: Procedure: Allogeneic stem cell transplantation
Drug: Fludarabine monophosphate
Drug: Busulfan
Drug: Bendamustine

First Posted Date: 2016-06-14

Last Posted Date: 2020-11-23

Lead Sponsor: St. Petersburg State Pavlov Medical University

Target Recruit Count: 27

Registration Number: NCT02799147

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

HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

Phase 2

Completed

Conditions: Myelodysplastic Syndrome (MDS)
Chronic Lymphocytic Leukemia (CLL)
Chemotherapy-sensitive Lymphoma
Acute Lymphoblastic Leukemia (ALL)/T Lymphoblastic Lymphoma
Acute Myelogenous Leukemia (AML)
Acute Biphenotypic Leukemia (ABL)
Acute Undifferentiated Leukemia (AUL)

Interventions: Drug: Fludarabine
Drug: Cyclophosphamide 14.5 mg/kg/day IV on Days -6, -5
Drug: Cyclophosphamide 50mg/kg/day IV on Days -5,-4
Radiation: Total Body Irradiation (TBI) 200cGy on Day -1
Drug: Cyclophosphamide 50mg/kg/day IV on Days -2,-1
Drug: Busulfan
Radiation: Total Body Irradiation (TBI) 200cGy twice a day on Days -3, -2, -1
Procedure: Infusion of non-T-cell depleted bone marrow on Day 0
Drug: Post-HCT Cyclophosphamide 50mg/kg IV on Day+3, +4
Drug: Sirolimus
Drug: Mycophenolate mofetil
Drug: G-CSF
Drug: Pre-HCT Mesna on Days -2 and -1
Drug: Pre-HCT Mesna on Days -5 and -4
Drug: Pre-HCT Mesna on Days -6 and -5
Drug: Post-HCT Mesna

First Posted Date: 2016-06-08

Last Posted Date: 2020-12-02

Lead Sponsor: Center for International Blood and Marrow Transplant Research

Target Recruit Count: 80

Registration Number: NCT02793544

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center - Adults, New York, New York, United States
🇺🇸
Karmanos Cancer Institute, Detroit, Michigan, United States
🇺🇸
Virginia Commonwealth University Massey Cancer Center Bone Marrow Transplant Program, Richmond, Virginia, United States

and more 8 locations

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

Phase 2

Completed

Conditions: Sickle Cell Disease

Interventions: Procedure: Standard of Care
Drug: Busulfan
Drug: Fludarabine
Drug: r-ATG
Procedure: Hematopoietic Cell Transplant
Drug: Tacrolimus
Drug: Methotrexate
Drug: Alemtuzumab
Drug: Total Body Irradiation (TBI)
Drug: Sirolimus
Drug: Melphalan
Drug: G-CSF

First Posted Date: 2016-05-09

Last Posted Date: 2024-04-17

Lead Sponsor: Medical College of Wisconsin

Target Recruit Count: 138

Registration Number: NCT02766465

Locations: 🇺🇸
Dana Farber Cancer Institute/Brigham & Women's Hospital, Boston, Massachusetts, United States
🇺🇸
Boston University, Boston, Massachusetts, United States
🇺🇸
Dana Farber Cancer Institute/Massachusetts General Hospital, Boston, Massachusetts, United States

and more 32 locations

Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation

Phase 1

Terminated

Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndrome
Acute Lymphocytic Leukemia

Interventions: Drug: Fludarabine
Radiation: Fractionated total body irradiation
Drug: Busulfan
Drug: Cyclophosphamide
Radiation: Single dose total body irradiation
Drug: Melphalan
Drug: Granulocyte-colony stimulating factor
Procedure: Stem cell transplant
Drug: Azacitidine

First Posted Date: 2016-04-25

Last Posted Date: 2020-10-20

Lead Sponsor: Washington University School of Medicine

Target Recruit Count: 5

Registration Number: NCT02750254

Locations: 🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States

Personalized NK Cell Therapy in CBT

Phase 2

Recruiting

Conditions: Acute Myeloid Leukemia With Myelodysplasia-Related Changes
Acute Lymphoblastic Leukemia
ISS Stage II Plasma Cell Myeloma
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
Acute Biphenotypic Leukemia
B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1
Chemotherapy-Related Leukemia
High Grade B-Cell Lymphoma With MYC and BCL2 or BCL6 Rearrangements
ISS Stage III Plasma Cell Myeloma
Chronic Myelomonocytic Leukemia

Interventions: Biological: Allogeneic Natural Killer Cell Line NK-92
Biological: Anti-Thymocyte Globulin
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Clofarabine
Drug: Fludarabine Phosphate
Drug: Melphalan
Other: Laboratory Biomarker Analysis
Biological: Rituximab
Radiation: Total-Body Irradiation
Procedure: Umbilical Cord Blood Transplantation

First Posted Date: 2016-04-05

Last Posted Date: 2024-06-10

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 100

Registration Number: NCT02727803

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia

Phase 3

Recruiting

Conditions: Acute Myeloid Leukaemia

Interventions: Drug: Mitoxantrone
Drug: Liposomal daunorubicin
Drug: Gemtuzumab ozogamicin
Drug: Fludarabine
Drug: Cytarabine
Drug: Busulfan
Drug: Cyclophosphamide

First Posted Date: 2016-03-31

Last Posted Date: 2021-10-08

Lead Sponsor: University of Birmingham

Target Recruit Count: 700

Registration Number: NCT02724163

Locations: 🇦🇺
Monash Children's Hospital, Melbourne, Australia
🇦🇺
Women and Children's Hospital Adelaide, Adelaide, Australia
🇦🇺
Queensland Children's Hospital, Brisbane, Australia

and more 65 locations

Belinostat Combined With Azacitidine/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Refractory or Relapsed Lymphoma

Phase 1

Withdrawn

Conditions: Lymphoma

Interventions: Drug: Busulfan
Drug: Caphosol
Drug: Glutamine
Drug: Pyridoxine
Drug: Belinostat
Drug: Azacitidine
Drug: Gemcitabine
Drug: Melphalan
Procedure: Stem Cell Transplant

First Posted Date: 2016-03-08

Last Posted Date: 2016-05-04

Lead Sponsor: M.D. Anderson Cancer Center

Registration Number: NCT02701673

Autologous Hematopoietic Stem Cell Transplantation as Adjuvant Treatment for Triple Negative Breast Cancer Patients

Phase 2

Conditions: Triple-Negative Invasive Breast Carcinoma
Residual Tumor

Interventions: Drug: Carmustine
Drug: Cyclophosphamide
Drug: Carboplatin
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Drug: Busulfan

First Posted Date: 2016-02-01

Last Posted Date: 2020-01-14

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

Target Recruit Count: 20

Registration Number: NCT02670109

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

URMC Related Haplo-identical Donor BMT

Phase 1

Completed

Conditions: Immune Deficiencies
Solid Tumors
Myelofibrosis
Multiple Myeloma
Lymphoma
Hematological Disease

Interventions: Radiation: Total Body Irradiation 1200 cGy
Drug: Fludarabine
Drug: Melphalan
Drug: Pre-Stem Cell Infusion Cyclophosphamide
Procedure: Stem Cell Infusion
Drug: Pre-Stem Cell Infusion Mesna
Drug: Post-Stem Cell Infusion Cyclophosphamide
Drug: Busulfan
Drug: Post-Stem Cell Infusion Mesna
Drug: Thiotepa

First Posted Date: 2016-01-21

Last Posted Date: 2021-11-03

Lead Sponsor: University of Rochester

Target Recruit Count: 74

Registration Number: NCT02660281

Locations: 🇺🇸
Wilmot Cancer Institute, Rochester, New York, United States

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