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    DRUG:

    busulfan

    Company:
    Generic mfg.
    Drug class:
    Alkylating agent
    Related drugs:
    1d
    Model-based antithymocyte globulin dosing in ex vivo CD34+ selected allogeneic haematopoietic cell transplantation: a single-centre, single-arm, phase 2 study. (PubMed, Lancet Haematol)
    These results demonstrate that model-based ATG dosing promotes robust CD4+ immune reconstitution after ex vivo CD34+ selected allogeneic HCT, underscoring the potential of pharmacokinetically guided ATG as a strategy to optimise immune recovery in myeloablative, calcineurin inhibitor-free transplantation for haematological malignancies.
    P2 data • Preclinical • Journal
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    CD34 (CD34 molecule)
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    cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
    2d
    Auto BMT for Non-M3 AML in 1st Remission in Pts </=60y of Age Using Busulfan/FTBI & VP16 as a Prep R (clinicaltrials.gov)
    P2, N=60, Completed, City of Hope Medical Center | Active, not recruiting --> Completed
    Trial completion
    |
    cytarabine • etoposide IV • idarubicin hydrochloride • Proleukin (aldesleukin) • busulfan • Neupogen (filgrastim)
    4d
    Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus (clinicaltrials.gov)
    P1/2, N=0, Withdrawn, National Institute of Allergy and Infectious Diseases (NIAID) | N=30 --> 0 | Trial completion date: Sep 2026 --> Nov 2025 | Enrolling by invitation --> Withdrawn | Trial primary completion date: Sep 2026 --> Nov 2025
    Enrollment change • Trial completion date • Trial withdrawal • Trial primary completion date
    |
    JAK3 (Janus Kinase 3)
    |
    sirolimus • busulfan
    6d
    Quantitative proteomic analysis of pathological and physiological ovarian aging: model evaluation, molecular mechanisms, and identification of early biomarkers and therapeutic targets. (PubMed, J Ovarian Res)
    This study confirmed the validity of the cyclophosphamide-busulfan induced mouse model of ovarian senescence, highlighted both the shared and distinct molecular mechanisms of physiological and pathological ovarian aging, and identified promising early biomarkers and therapeutic intervention targets.
    Journal
    |
    GSTP1 (Glutathione S-transferase pi 1) • CYP17A1 (Cytochrome P450 Family 17 Subfamily A Member 1) • SDHD (Succinate Dehydrogenase Complex Subunit D) • LRP4 (LDL Receptor Related Protein 4)
    |
    cyclophosphamide • busulfan
    8d
    Gene Therapy After Frontline Chemotherapy in Treating Patients With AIDS-Related Non-Hodgkin Lymphoma (clinicaltrials.gov)
    P1, N=3, Active, not recruiting, City of Hope Medical Center | Trial completion date: Aug 2025 --> Aug 2026 | Trial primary completion date: Aug 2025 --> Aug 2026
    Trial completion date • Trial primary completion date
    |
    CD34 (CD34 molecule)
    |
    busulfan
    14d
    CSIDE: Conditioning SCID Infants Diagnosed Early (clinicaltrials.gov)
    P2, N=56, Active, not recruiting, Center for International Blood and Marrow Transplant Research | Recruiting --> Active, not recruiting | Trial completion date: Aug 2030 --> Dec 2028 | Trial primary completion date: Aug 2029 --> Dec 2027
    Enrollment closed • Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • JAK3 (Janus Kinase 3) • HLA-B (Major Histocompatibility Complex, Class I, B) • CD31 (Platelet and endothelial cell adhesion molecule 1) • PECAM1 (Platelet And Endothelial Cell Adhesion Molecule 1) • HLA-C (Major Histocompatibility Complex, Class I, C) • RAG1 (Recombination Activating 1) • IL2RG (Interleukin 2 Receptor Subunit Gamma)
    |
    busulfan
    16d
    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) (clinicaltrials.gov)
    P3, N=340, Recruiting, Merck Sharp & Dohme LLC | Trial primary completion date: Sep 2026 --> Jul 2027
    Trial primary completion date
    |
    Jakafi (ruxolitinib) • hydroxyurea • busulfan • bomedemstat (MK-3543)
    17d
    Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov)
    P2, N=22, Active, not recruiting, Masonic Cancer Center, University of Minnesota | Completed --> Active, not recruiting | Trial completion date: Dec 2023 --> Dec 2026
    Enrollment closed • Trial completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • NPM1 (Nucleophosmin 1) • KMT2A (Lysine Methyltransferase 2A) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha)
    |
    FLT3-ITD mutation • NPM1 mutation • MLL rearrangement
    |
    cyclophosphamide • melphalan • fludarabine IV • busulfan • spanlecortemlocel (MGTA-456)
    18d
    Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency (clinicaltrials.gov)
    P2, N=70, Active, not recruiting, National Cancer Institute (NCI) | Recruiting --> Active, not recruiting | N=128 --> 70 | Trial completion date: Dec 2026 --> Oct 2028 | Trial primary completion date: Dec 2025 --> Oct 2026
    Enrollment closed • Enrollment change • Trial completion date • Trial primary completion date
    |
    DOCK8 (Dedicator Of Cytokinesis 8)
    |
    cyclophosphamide • fludarabine IV • busulfan
    21d
    Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes (clinicaltrials.gov)
    P2, N=50, Not yet recruiting, Fred Hutchinson Cancer Center
    New P2 trial
    |
    Jakafi (ruxolitinib) • cyclophosphamide • methotrexate • melphalan • fludarabine IV • busulfan • methotrexate IV
    22d
    Allogeneic transplantation in adverse-risk acute myeloid leukemia: Challenges, strategies, and future directions. (PubMed, Chin J Cancer Res)
    Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the cornerstone of consolidation for eligible patients; however, due to high relapse rates, regimen-related toxicity, and variable responses across genetic subtypes, optimal transplant timing, conditioning regimens (e.g., busulfan- or melphalan-based protocols), and bridging strategies require further refinement. Meanwhile, post-transplant maintenance therapies, such as hypomethylating agents or targeted drugs, are one emerging area under investigation for relapse prevention. In this perspective, we review the latest advances in allo-HSCT strategies for adverse-risk AML and highlight the importance of molecularly-guided approaches to improve outcomes in these aggressive subtypes.
    Journal
    |
    TP53 (Tumor protein P53) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NUP214 (Nucleoporin 214) • FUS (FUS RNA Binding Protein)
    |
    TP53 mutation
    |
    melphalan • busulfan
    22d
    RAFA: HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (clinicaltrials.gov)
    P2, N=70, Recruiting, Children's Hospital Medical Center, Cincinnati | Trial completion date: Jul 2027 --> Dec 2028 | Trial primary completion date: Jul 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
    |
    cyclophosphamide • fludarabine IV • busulfan • Neupogen (filgrastim)