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

Advanced Drug Monograph

Access comprehensive pharmacological analysis, clinical applications, and therapeutic considerations.

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

Base Editing Hematopoietic Stem Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

Phase 1

Conditions: CD40L-HyperIgM Syndrome

Interventions: Drug: Busulfan
Drug: Palifermin
Drug: Sirolimus
Drug: Alemtuzumab
Biological: Base-edited hematopoietic stem and progenitor cells

First Posted Date: 2025-05-07

Last Posted Date: 2025-05-21

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

Target Recruit Count: 1

Registration Number: NCT06959771

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

Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial)

Phase 2

Not yet recruiting

Conditions: Acute Myeloid Leukemia

Interventions: Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
Procedure: Biospecimen Collection
Procedure: Bone Marrow Biopsy
Drug: Busulfan
Procedure: Chest Radiography
Procedure: Computed Tomography
Procedure: Echocardiography Test
Drug: Fludarabine
Drug: Melphalan
Procedure: Multigated Acquisition Scan
Drug: Placebo Administration
Procedure: Positron Emission Tomography
Radiation: Total-Body Irradiation
Drug: Venetoclax

First Posted Date: 2025-05-02

Last Posted Date: 2025-05-11

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 244

Registration Number: NCT06954987

Allo HSCT for High Risk Hemoglobinopathies

Phase 2

Recruiting

Conditions: Graft Failure
Sickle Cell Disease
Hemoglobinopathies

Interventions: Drug: Alemtuzumab
Radiation: Total Body Irradiation
Drug: Thymoglobulin
Biological: Cell Infusion
Drug: Fludarabine
Drug: Sirolimus
Drug: Busulfan
Drug: Tacrolimus
Drug: Thiotepa
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil

First Posted Date: 2025-03-12

Last Posted Date: 2025-03-12

Lead Sponsor: Masonic Cancer Center, University of Minnesota

Target Recruit Count: 62

Registration Number: NCT06872333

Locations: 🇺🇸
Masonic Cancer Center, Minneapolis, Minnesota, United States

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

Phase 1

Conditions: X-linked Severe Combined Immunodeficiency
X-SCID
XSCID

Interventions: Genetic: Plerixafor
Drug: Filgrastim
Drug: Palifermin
Drug: Busulfan
Biological: Base-edited hematopoietic stem and progenitor cells

First Posted Date: 2025-02-28

Last Posted Date: 2025-05-18

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

Target Recruit Count: 18

Registration Number: NCT06851767

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

Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation

Phase 1

Recruiting

Conditions: Hematologic Diseases

Interventions: Drug: Busulfan (Busulfex)
Drug: Cyclophosphamide (CTX)
Drug: Fludarabine (Fludara)
Drug: Semustine (MeCCNU)

First Posted Date: 2025-02-05

Last Posted Date: 2025-02-05

Lead Sponsor: He Huang

Target Recruit Count: 29

Registration Number: NCT06809699

Locations: 🇨🇳
The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China

Human Leukocyte Antigen (HLA) Mismatched Unrelated Allogeneic Hematopoietic Stem Cell Transplantation

Phase 1

Recruiting

Conditions: Hematologic Disease

Interventions: Drug: Busulfan (Busulfex)
Drug: Cyclophosphamide (CTX)
Drug: Fludarabine (Fludara)
Drug: Semustine (MeccNU)

First Posted Date: 2025-02-05

Last Posted Date: 2025-02-05

Lead Sponsor: He Huang

Target Recruit Count: 29

Registration Number: NCT06809712

Locations: 🇨🇳
The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China

Intensity Modulated Total Marrow Irradiation in Fully Human Leukocyte Antigen (HLA)-Matched and Partially-HLA Mismatched Allogeneic Transplantation Patients with High-Risk Acute Myeloid Leukemia (AML), Chronic Myeloid Leukemia (CML), and Myelodysplastic Syndrome (MDS)

Phase 2

Recruiting

Conditions: Acute Myeloid Leukemia, Relapsed, Adult
Acute Myeloid Leukemia Refractory
Chronic Myeloid Leukemia - Accelerated Phase
Myelodysplastic Syndromes

Interventions: Radiation: Intensity modulated total marrow irradiation
Drug: Cyclophosphamide (CTX)
Drug: Fludarabine (Fludara)
Drug: Busulfan (conditioning for ALLO Transplant)

First Posted Date: 2025-01-31

Last Posted Date: 2025-02-04

Lead Sponsor: University of Illinois at Chicago

Target Recruit Count: 38

Registration Number: NCT06802315

Locations: 🇺🇸
University of Illinois Cancer Center, Chicago, Illinois, United States

Twice-per-weekSelinexor, 2 Days Melphalan

Phase 3

Not yet recruiting

Conditions: Acute Myeloid Leukemia

Interventions: Drug: Selinexor
Drug: Melphalan
Drug: Intravenous Busulfan
Drug: Fludarabine

First Posted Date: 2024-09-25

Last Posted Date: 2024-09-25

Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Target Recruit Count: 126

Registration Number: NCT06613035

Locations: 🇨🇳
Zhengzhou University First Affiliated Hospital, Zhengzhou, Henan, China
🇨🇳
Hebei Medical University Second Hospital, Shijiazhuang, hebeisheng, China
🇨🇳
The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army, Jinan, Shandong, China

and more 1 locations