MedPath

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

Adding Venetoclax to the High-dose Chemotherapy Regimen Prior to Mismatche Allogeneic Stem Cell Transplant

Phase 2
Withdrawn
Conditions
Myelodysplastic Syndromes
Acute Myeloid Leukemia
Interventions
First Posted Date
2024-03-29
Last Posted Date
2024-06-14
Lead Sponsor
Northside Hospital, Inc.
Registration Number
NCT06337331
Locations
🇺🇸

Caitlin Guzowski, Atlanta, Georgia, United States

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

Phase 1
Suspended
Conditions
Chronic Granulomatous Disease (CGD)
X-Linked Chronic Granulomatous Disease
Interventions
Biological: Base-edited hematopoietic stem and progenitor cells
First Posted Date
2024-03-22
Last Posted Date
2025-05-16
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Target Recruit Count
1
Registration Number
NCT06325709
Locations
🇺🇸

National Institutes of Health Clinical Center, Bethesda, Maryland, United States

Compare the Efficacy and Safety of Dec-FB4 and FB4 as Conditioning Regimen for AML-MR

Phase 3
Not yet recruiting
Conditions
AML, Adult
Interventions
First Posted Date
2024-03-07
Last Posted Date
2024-06-04
Lead Sponsor
Ruijin Hospital
Target Recruit Count
220
Registration Number
NCT06297772
Locations
🇨🇳

Ruijin Hospital, Shanghai, China

Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation

Phase 2
Recruiting
Conditions
Hematologic Malignancy
Graft Versus Host Disease
Interventions
Drug: Cycophosphamide
Drug: Anti-Thymoglobulin
Other: Donor Lymphocytes Injection
Radiation: total body irradiation
Other: hematopoietic stem cells
Other: Graft nuclear cells
First Posted Date
2024-02-12
Last Posted Date
2024-10-28
Lead Sponsor
Nantes University Hospital
Target Recruit Count
82
Registration Number
NCT06252870
Locations
🇫🇷

CHU Angers, Angers, France

🇫🇷

CHU Brest, Brest, France

🇫🇷

CHU Nantes, Nantes, France

Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy with Elranatamab in Patients with High-risk Multiple Myeloma

Phase 2
Recruiting
Conditions
Post-transplant MRD-guided Maintenance Therapy
Multiple Myeloma
Pre-transplant Purging
Interventions
First Posted Date
2024-01-17
Last Posted Date
2025-02-13
Lead Sponsor
M.D. Anderson Cancer Center
Target Recruit Count
40
Registration Number
NCT06207799
Locations
🇺🇸

MD Anderson Cancer Center, Houston, Texas, United States

Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML

Phase 1
Recruiting
Conditions
Aml
Acute Myeloid Leukemia, Pediatric
Acute Myeloid Leukemia
AML, Childhood
Interventions
Biological: Granulocyte Colony-Stimulating Factor
Biological: TCR alpha beta / CD19+ depleted haploidentical hematopoietic progenitor cell graft
Biological: memory-like natural killer cells
Device: CliniMACS
First Posted Date
2023-12-06
Last Posted Date
2025-05-20
Lead Sponsor
Washington University School of Medicine
Target Recruit Count
48
Registration Number
NCT06158828
Locations
🇺🇸

Washington University School of Medicine, Saint Louis, Missouri, United States

A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)

Phase 3
Recruiting
Conditions
Essential Thrombocythemia
Interventions
Drug: Interferon alfa/pegylated interferon alfa
First Posted Date
2023-10-12
Last Posted Date
2025-05-16
Lead Sponsor
Merck Sharp & Dohme LLC
Target Recruit Count
300
Registration Number
NCT06079879
Locations
🇺🇸

Los Angeles Cancer Network ( Site 3491), Glendale, California, United States

🇺🇸

Stanford Cancer Institute ( Site 0107), Stanford, California, United States

🇺🇸

The Lundquist Institute ( Site 3423), Torrance, California, United States

and more 155 locations

The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

Phase 4
Recruiting
Conditions
Severe Aplastic Anemia
Interventions
Drug: Flu/Cy/ATG or Cy/ATG
First Posted Date
2023-10-05
Last Posted Date
2023-11-18
Lead Sponsor
Peking University People's Hospital
Target Recruit Count
160
Registration Number
NCT06069180
Locations
🇨🇳

Deparment of Hematology, Peking University People's Hospital, Beijing, Beijing, China

HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis

Phase 2
Recruiting
Conditions
Acute Myeloid Leukemia
Myelodysplastic Syndromes
Chronic Myeloid Leukemia
Chronic Lymphocytic Leukemia
Chronic Myelomonocytic Leukemia
Acute Leukemia
Pro-Lymphocytic Leukemia
Acute Lymphoblastic Leukemia
Myelofibrosis
Myeloproliferative Neoplasm
Interventions
Procedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT)
Other: Patient Reported Outcomes
Radiation: Total-body irradiation
First Posted Date
2023-08-21
Last Posted Date
2025-04-03
Lead Sponsor
Center for International Blood and Marrow Transplant Research
Target Recruit Count
190
Registration Number
NCT06001385
Locations
🇺🇸

Honor Health, Scottsdale, Arizona, United States

🇺🇸

University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States

🇺🇸

Mayo Clinic Jacksonville, Jacksonville, Florida, United States

and more 25 locations

Study Comparing the Efficacy of 2 RIC Regimens (Clofarabine vs Fludarabine) in Adults With AML Eligible to Allo-SCT

Phase 2
Recruiting
Conditions
Acute Myeloid Leukemia in Remission
Interventions
First Posted Date
2023-06-23
Last Posted Date
2023-11-15
Lead Sponsor
Nantes University Hospital
Target Recruit Count
302
Registration Number
NCT05917405
Locations
🇫🇷

CRLC Caen, Caen, France

🇫🇷

CHU Limoges, Limoges, France

🇫🇷

CHU Besançon, Besançon, France

and more 20 locations
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