^
    Contact us  to learn more about
    our Premium Content:  News alerts, weekly reports and conference planners
    DRUG:

    Grafapex (treosulfan)

    i
    Other names: NSC 39069, CB 2562, CB 40067, CCRIS 2781, HSDB 6963
    Company:
    Medac, Medexus
    Drug class:
    Alkylating agent
    Related drugs:
    6d
    MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases (clinicaltrials.gov)
    P2, N=132, Recruiting, Masonic Cancer Center, University of Minnesota | Not yet recruiting --> Recruiting | Initiation date: Aug 2026 --> Apr 2026
    Enrollment open • Trial initiation date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cyclophosphamide • fludarabine IV • Grafapex (treosulfan)
    21d
    Successful Haplo-Hematopoietic Stem Cell Transplantation for Juvenile Myelomonocytic Leukemia in a Child With Underlying Thrombocytopenia-Absent Radius Syndrome: A Unique Case. (PubMed, Cancer Rep (Hoboken))
    This case represents, to our knowledge, one of the very few-if not the first-reported instances of successful HSCT for JMML in a patient with TAR syndrome. It underscores the importance of vigilant surveillance in TAR patients for potential malignant transformation and demonstrates the curative potential of HSCT in rare congenital-hematologic overlap syndromes.
    Journal
    |
    NF1 (Neurofibromin 1) • RBM8A (RNA Binding Motif Protein 8A)
    |
    RAS mutation
    |
    cyclophosphamide • melphalan • Grafapex (treosulfan)
    28d
    MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases (clinicaltrials.gov)
    P2, N=132, Not yet recruiting, Masonic Cancer Center, University of Minnesota
    New P2 trial
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    cyclophosphamide • fludarabine IV • Grafapex (treosulfan)
    1m
    Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders (clinicaltrials.gov)
    P2, N=40, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Jul 2027 --> Jul 2028 | Trial primary completion date: Jul 2027 --> Jul 2028
    Trial completion date • Trial primary completion date
    |
    CD33 (CD33 Molecule) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    fludarabine IV • thiotepa • busulfan • Grafapex (treosulfan)
    1m
    RESOLVE trial: Registry and clinical trial comparing standard intensity and reduced intensity treatment in patients with AML or CLL who do not have residual disease. (2024-512503-39-00)
    P4, N=289, Recruiting, Medizinische Hochschule Hannover | N=128 --> 289
    Enrollment change • Minimal residual disease
    |
    Venclexta (venetoclax) • Imbruvica (ibrutinib) • cytarabine • Gazyva (obinutuzumab) • cyclophosphamide • etoposide IV • midostaurin • Vanflyta (quizartinib) • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • idarubicin hydrochloride • mitoxantrone • cladribine • fludarabine IV • thiotepa • busulfan • Grafapex (treosulfan)
    1m
    Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) (clinicaltrials.gov)
    P2, N=40, Completed, Fred Hutchinson Cancer Center | Active, not recruiting --> Completed
    Trial completion
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-DQB1 (Major Histocompatibility Complex, Class II, DQ Beta 1) • GATA2 (GATA Binding Protein 2) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    LDH elevation
    |
    methotrexate • fludarabine IV • busulfan • Grafapex (treosulfan) • methotrexate IV
    2ms
    RESOLVE trial: Registry and clinical trial comparing standard intensity and reduced intensity treatment in patients with AML or CLL who do not have residual disease. (2024-512503-39-00)
    P4, N=128, Recruiting, Medizinische Hochschule Hannover | N=52 --> 128
    Enrollment change • Minimal residual disease
    |
    Venclexta (venetoclax) • Imbruvica (ibrutinib) • cytarabine • Gazyva (obinutuzumab) • cyclophosphamide • etoposide IV • midostaurin • Vanflyta (quizartinib) • Mylotarg (gemtuzumab ozogamicin) • daunorubicin • idarubicin hydrochloride • mitoxantrone • cladribine • fludarabine IV • thiotepa • busulfan • Grafapex (treosulfan)
    2ms
    PKBMT: Precision Dosing of Anti-Cancer Medicines for patients undergoing Oncology, Blood Transplant and Cellular Therapies. (ACTRN12626000285358)
    P=N/A, N=300, Not yet recruiting, The Children's Hospital at Westmead
    New trial
    |
    fludarabine IV • Grafapex (treosulfan)
    2ms
    Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies (clinicaltrials.gov)
    P2, N=60, Recruiting, Fred Hutchinson Cancer Center | Trial completion date: Jan 2028 --> Mar 2029 | Trial primary completion date: Feb 2026 --> Apr 2027
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
    |
    cyclophosphamide • fludarabine IV • busulfan • cyclosporin A microemulsion • Grafapex (treosulfan) • Neupogen (filgrastim)
    3ms
    Allogeneic HSCT for consolidation in pediatric refractory or relapsed ALK-positive anaplastic large cell lymphoma. (PubMed, Blood Adv)
    Conditioning was based on total body irradiation (TBI) in 30 patients and on chemotherapy in 27 patients, mainly with reduced-toxicity conditioning (RTC; Treosulfan, Fludarabine, and Thiotepa). Our data support the use of TBI-free conditioning and suggest improved outcomes with unrelated donors receiving ATLG prophylaxis. NCT00317408.
    Journal
    |
    ALK (Anaplastic lymphoma kinase)
    |
    ALK positive
    |
    fludarabine IV • thiotepa • Grafapex (treosulfan)
    3ms
    Allogeneic Hematopoietic Stem Cell Transplantation in ERCC6L2 Disease. (PubMed, Blood Adv)
    The use of non-treosulfan-based myeloablative conditioning (MAC) regimens increased the risk of endothelial complications compared to reduced-intensity conditioning (RIC) (HR, 4.9; 95% CI, 1.1-22.0; P=0.040), whereas outcomes with treosulfan-based MAC were comparable to RIC. In summary, allogeneic HSCT is a viable curative strategy for ED when performed before transformation to an aggressive malignancy i.e. myelodysplasia with excess blasts or acute myeloid leukemia. However, the elevated incidence of endothelial toxicity highlights the importance of optimizing conditioning intensity and enhancing peritransplant monitoring in this population.
    Clinical • Journal
    |
    TP53 (Tumor protein P53) • ERCC6 (Excision repair cross-complementation group 6)
    |
    TP53 mutation
    |
    Grafapex (treosulfan)
    4ms
    Study of Treosulfan-Based Conditioning for HSCT in Nijmegen Breakage Syndrome (clinicaltrials.gov)
    P2, N=24, Not yet recruiting, Federal Research Institute of Pediatric Hematology, Oncology and Immunology
    New P2 trial
    |
    Grafapex (treosulfan)