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 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

Gene Editing For Sickle Cell Disease

First Posted Date
2024-07-17
Last Posted Date
2024-12-16
Lead Sponsor
St. Jude Children's Research Hospital
Target Recruit Count
25
Registration Number
NCT06506461
Locations
🇺🇸

St. Jude Children's Research Hospital, Memphis, Tennessee, United States

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

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

First Posted Date
2024-03-22
Last Posted Date
2024-12-19
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Target Recruit Count
10
Registration Number
NCT06325709
Locations
🇺🇸

National Institutes of Health Clinical Center, Bethesda, Maryland, 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)

First Posted Date
2023-10-12
Last Posted Date
2024-12-06
Lead Sponsor
Merck Sharp & Dohme LLC
Target Recruit Count
300
Registration Number
NCT06079879
Locations
🇨🇳

Anhui Provincial Hospital ( Site 3513), Hefei, Anhui, China

🇨🇳

Peking University Third Hospital-Hematology ( Site 3502), Beijing, Beijing, China

🇨🇳

The Second Affiliated Hospital Of Fujian Medical University ( Site 3525), Quanzhou, Fujian, China

and more 130 locations

The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

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

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