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 t...

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...

Associated Conditions: Chronic Myelogenous Leukemia (CML), Essential Thrombocythemia (ET), Polycythemia Vera (PV)

Associated Therapies: Conditioning regimens for allogeneic stem cell transplantation therapy

Clinical Trials

Optimizing PTCy Dose and Timing

Phase 1

Recruiting

Conditions: Hematologic Neoplasms
Graft Versus Host Disease

Interventions: Drug: Busulfan
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Drug: Sirolimus

First Posted Date: 2019-06-12

Last Posted Date: 2024-11-25

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 400

Registration Number: NCT03983850

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

Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)

First Posted Date: 2019-05-31

Last Posted Date: 2024-08-22

Lead Sponsor: Fred Hutchinson Cancer Center

Target Recruit Count: 120

Registration Number: NCT03970096

Locations: 🇺🇸
University of Pittsburgh Cancer Institute (UPCI), Pittsburgh, Pennsylvania, United States
🇺🇸
Moffitt Cancer Center, Tampa, Florida, United States
🇺🇸
Fred Hutch/University of Washington Cancer Consortium, Seattle, Washington, United States

Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide

Early Phase 1

Recruiting

Conditions: Chronic Granulomatous Disease

Interventions: Drug: Busulfan
Drug: Alemtuzumab
Drug: Cyclophosphamide
Drug: Sirolimus
Radiation: Total Body Irradiation
Biological: Allogeneic peripheral blood stem cell

First Posted Date: 2019-04-10

Last Posted Date: 2024-10-30

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

Target Recruit Count: 30

Registration Number: NCT03910452

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

BuCE Versus BuME as Conditioning Therapy in Non-Hodgkin's Lymphoma

Phase 2

Completed

Conditions: Non-hodgkin Lymphoma

Interventions: Drug: Busulfan
Drug: Cyclophosphamide
Drug: Etoposide
Drug: Melphalan

First Posted Date: 2019-01-04

Last Posted Date: 2019-01-07

Lead Sponsor: Soonchunhyang University Hospital

Target Recruit Count: 75

Registration Number: NCT03794167

Locations: 🇰🇷
Jong-Ho Won, Seoul, Korea, Republic of

Busulfan, Melphalan and Etoposide as Conditioning for Autologous Transplantation for Non-Hodgkin's Lymphoma (NHL)

Phase 2

Completed

Conditions: Non-Hodgkin's Lymphoma

Interventions: Drug: Busulfan
Drug: Melphalan
Drug: Etoposide

First Posted Date: 2019-01-03

Last Posted Date: 2019-01-04

Lead Sponsor: Soonchunhyang University Hospital

Target Recruit Count: 51

Registration Number: NCT03792815

Locations: 🇰🇷
Soonchunhyang University Hospital, Seoul, Korea, Republic of

Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell Transplant

First Posted Date: 2018-12-19

Last Posted Date: 2024-11-14

Lead Sponsor: Fred Hutchinson Cancer Center

Target Recruit Count: 68

Registration Number: NCT03779854

Locations: 🇺🇸
Children's Hospital of Los Angeles, Los Angeles, California, United States
🇺🇸
Children's National Medical Center, Washington, District of Columbia, United States
🇺🇸
Children's Healthcare of Atlanta, Atlanta, Georgia, United States

and more 7 locations

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Phase 1

Completed

Conditions: Severe Combined Immunodeficiency Due to ADA Deficiency

Interventions: Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101)
Drug: Busulfan
Drug: Peg-Ada

First Posted Date: 2018-12-05

Last Posted Date: 2023-02-01

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

Target Recruit Count: 13

Registration Number: NCT03765632

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

A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

Phase 1

Active, not recruiting

Conditions: Sickle Cell Disease

Interventions: Biological: Plerixafor
Drug: Busulfan
Genetic: BIVV003

First Posted Date: 2018-08-31

Last Posted Date: 2023-11-21

Lead Sponsor: Sangamo Therapeutics

Target Recruit Count: 8

Registration Number: NCT03653247

Locations: 🇺🇸
UCSF Benioff Children's Hospital, Oakland, California, United States
🇺🇸
Investigational Site Number 101, Bethesda, Maryland, United States
🇺🇸
Karmanos Cancer Institute, Detroit, Michigan, United States

and more 2 locations

Conditioning SCID Infants Diagnosed Early

Phase 2

Recruiting

Conditions: SCID

Interventions: Drug: Busulfan
Device: Cell processing for TCRαβ+/CD19+ depletion

First Posted Date: 2018-08-08

Last Posted Date: 2023-09-18

Lead Sponsor: Michael Pulsipher, MD

Target Recruit Count: 64

Registration Number: NCT03619551

Locations: 🇺🇸
UCLA Center for Health Sciences, Los Angeles, California, United States
🇺🇸
Lucile Packard Children's Hospital / Stanford Children's Health, Stanford, California, United States
🇺🇸
Nemours Alfred I. duPont Hospital for Children, Wilmington, Delaware, United States

and more 49 locations

Donor Stem Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors

Phase 2

Terminated

Conditions: Non-Hodgkin Lymphoma
Chronic Lymphocytic Leukemia
Acute Lymphoid Leukemia (ALL)
Chronic Myeloid Leukemia (CML)
Hodgkin Lymphoma
Acute Myeloid Leukemia (AML)

Interventions: Radiation: Hyperfractionated total body irradiation
Drug: Busulfan
Drug: Thiotepa
Drug: Fludarabine
Drug: Melphalan
Drug: Cyclophosphamide
Drug: Clofarabine
Procedure: HPC(A) stem cell allograft
Drug: Rituximab
Device: Rabbit antithymocyte globulin

First Posted Date: 2018-08-03

Last Posted Date: 2024-03-12

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 9

Registration Number: NCT03615105

Locations: 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States

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