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    DRUG:

    melphalan

    i
    Other names: L-PAM, L-sarcolysin, phenylalanine mustard
    Company:
    Generic mfg.
    Drug class:
    Alkylating agent
    Related drugs:
    17h
    Study of Thiotepa Combination With Melphalan (TM Protocol) Conditioning for Autologous Hematopoietic Stem Cell Transplantation in Multiple Myeloma：A Prospective Randomized Controlled Clinical Trial (clinicaltrials.gov)
    P=N/A, N=204, Not yet recruiting, First Affiliated Hospital Xi'an Jiaotong University
    New trial
    |
    CD34 (CD34 molecule)
    |
    melphalan • thiotepa
    2d
    CAR-T+ASCT: CAR-T Combined With ASCT in the Treatment of Relapsed/Refractory Large B-cell Lymphoma With High-risk Factors. (clinicaltrials.gov)
    P2, N=20, Not yet recruiting, Zhejiang Cancer Hospital
    New P2 trial
    |
    TP53 (Tumor protein P53) • BCL2 (B-cell CLL/lymphoma 2) • BCL6 (B-cell CLL/lymphoma 6)
    |
    TP53 mutation
    |
    cytarabine • Yescarta (axicabtagene ciloleucel) • carmustine • melphalan
    6d
    Therapy for Children With Advanced Stage Neuroblastoma (clinicaltrials.gov)
    P2, N=153, Completed, St. Jude Children's Research Hospital | Active, not recruiting --> Completed
    Trial completion
    |
    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
    |
    MYCN amplification
    |
    cisplatin • doxorubicin hydrochloride • cyclophosphamide • etoposide IV • vincristine • topotecan • melphalan • mesna • Proleukin (aldesleukin) • busulfan • Unituxin (dinutuximab) • Leukine (sargramostim) • Neupogen (filgrastim) • daretabart (hu1418K322A)
    6d
    UHKT-PTC-TMI-1: Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML/MDS (clinicaltrials.gov)
    P1/2, N=16, Recruiting, Institute of Hematology and Blood Transfusion, Czech Republic
    New P1/2 trial
    |
    cyclophosphamide • melphalan • fludarabine IV
    6d
    Adding Bortezomib and Bendamustine to High-Dose Melphalan in Autologous Haematopoietic Stem Cell Transplantation for Relapsed Multiple Myeloma-A Single Centre Retrospective Study. (PubMed, EJHaem)
    Compared with standard HDM, the BBM-protocol in ASCT2 was associated with a smaller decline in progression-free survival relative to the ASCT1, and a consistent trend toward higher effectiveness across other measures. These findings suggest that BBM may help preserve efficacy in the setting of repeated transplantation and underscore the need for larger prospective studies to confirm these results.
    Retrospective data • Journal
    |
    SLC1A5 (Solute Carrier Family 1 Member 5)
    |
    bortezomib • bendamustine • melphalan
    7d
    042011: LCH-IV International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis (2024-512676-36-00)
    P2/3, N=1124, Active, not recruiting, St. Anna Childrens Cancer Research Institute GmbH | Not yet recruiting --> Active, not recruiting
    Enrollment closed
    |
    cytarabine • methotrexate • cladribine • melphalan • fludarabine IV • mercaptopurine • vinblastine • prednisolone
    8d
    Amimestrocel Injection for Preventing Severe Oral Mucositis in HSCT Patients (clinicaltrials.gov)
    P2, N=22, Recruiting, The First Affiliated Hospital of Soochow University | Not yet recruiting --> Recruiting
    Enrollment open
    |
    cyclophosphamide • melphalan
    8d
    CD45RA-depleted CD19-CAR T Cell Consolidation After TCRαβ+/CD19 B Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma (clinicaltrials.gov)
    P1, N=60, Recruiting, St. Jude Children's Research Hospital | Initiation date: Mar 2026 --> Jun 2026
    Trial initiation date
    |
    cyclophosphamide • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)
    13d
    High Dose Melphalan: A pilot study to evaluate pharmacokinetic-based dose determination in patients with multiple myeloma scheduled to undergo autologous stem cell transplantation (ACTRN12613000487718)
    P4, N=30, Completed, The Children's Hospital at Westmead | Active, not recruiting --> Completed
    Trial completion
    |
    melphalan
    14d
    ALLOHA: A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation (clinicaltrials.gov)
    P1, N=75, Recruiting, TScan Therapeutics, Inc. | Trial primary completion date: Dec 2025 --> Jun 2026
    Trial primary completion date
    |
    HLA-A (Major Histocompatibility Complex, Class I, A)
    |
    melphalan • TSC-100 • TSC-101
    20d
    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)
    22d
    Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=59, Completed, City of Hope Medical Center | Active, not recruiting --> Completed | Trial completion date: Jan 2026 --> Oct 2025
    Trial completion • Trial completion date
    |
    sirolimus • melphalan • fludarabine IV • itacitinib (INCB039110)