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    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:
    15d
    OPTISAGE: Phase III Study Comparing GVHD Prophylaxis With ATG-thymoglobulin to ATLG-grafalon in Elderly Patients With Acute Myeloid Leukemia or Myelodysplasic Syndrome and Receiving an Allogeneic Hematopoietic Stem Cell Transplantation With a 10/10 HLA Matched Unrelated Donor (clinicaltrials.gov)
    P3, N=324, Recruiting, Assistance Publique - Hôpitaux de Paris | Trial primary completion date: Feb 2027 --> Nov 2028 | Not yet recruiting --> Recruiting
    Enrollment open • Trial primary completion date
    |
    fludarabine IV • Grafapex (treosulfan)
    18d
    Treosulfan Therapeutic Drug Monitoring in Pediatric Hematopoietic Stem Cell Transplant Recipients (clinicaltrials.gov)
    P=N/A, N=70, Recruiting, Fondazione IRCCS Policlinico San Matteo di Pavia | Trial completion date: Dec 2025 --> Jul 2027 | Trial primary completion date: Dec 2025 --> Jul 2027
    Trial completion date • Trial primary completion date
    |
    Grafapex (treosulfan)
    26d
    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)
    1m
    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)
    2ms
    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)
    2ms
    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)
    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=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)
    2ms
    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)