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    DRUG:

    clofarabine

    i
    Other names: JC0707, Cl-F-ara-A, CAFdA, GZ-393590, SAR-393590
    Company:
    Generic mfg.
    Drug class:
    DNA synthesis inhibitor
    Related drugs:
    2d
    US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov)
    P1/2, N=13, Completed, ExCellThera inc. | Recruiting --> Completed | Trial completion date: Jun 2027 --> Dec 2025 | Trial primary completion date: Jun 2026 --> Dec 2025
    Trial completion • Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
    |
    cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan • Zemcelpro (dorocubicel)
    19d
    Epigenetic remodeling via HDAC6 inhibition amplifies anti-tumoral immune responses in myeloid leukemia cells. (PubMed, Cell Death Dis)
    Moreover, an extended drug screening analysis identified Cytarabine and Clofarabine as significantly synergizing with HDAC6 inhibitor (Ricolinostat) in myeloid leukemia cell lines and in patient-derived xenograft (PDX) cells, while showing limited synergy in lymphoid leukemia cell lines, PDX, or healthy control cells. These findings suggest that HDAC6 represents a promising therapeutic target in myeloid lineage-derived leukemia cells by simultaneously enhancing immune activation and increasing chemosensitivity.
    Journal
    |
    CD8 (cluster of differentiation 8) • TNFA (Tumor Necrosis Factor-Alpha) • LAMP1 (Lysosomal Associated Membrane Protein 1) • HDAC6 (Histone Deacetylase 6)
    |
    cytarabine • clofarabine • rocilinostat (ACY-1215)
    26d
    Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related Disorders (clinicaltrials.gov)
    P2, N=25, Active, not recruiting, Dana-Farber Cancer Institute | Trial completion date: Jan 2026 --> Jul 2026
    Trial completion date
    |
    BRAF V600E • BRAF V600
    |
    clofarabine
    2ms
    A Prospective Clinical Study on the Efficacy and Safety of PEG-rhG-CSF in Preventing Neutropenia Induced by Clofarabine in Pediatric Patients with Langerhans Cell Histiocytosis (ChiCTR2500114943)
    P4, N=22, Not yet recruiting, West China Second University Hospital, Sichuan University; West China Second University Hospital, Sichuan University
    New P4 trial
    |
    clofarabine
    3ms
    Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders (clinicaltrials.gov)
    P2, N=31, Recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026
    Trial completion date • Trial primary completion date
    |
    KMT2A (Lysine Methyltransferase 2A)
    |
    MLL rearrangement
    |
    clofarabine • fludarabine IV • busulfan • cyclosporin A microemulsion
    4ms
    Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=72, Active, not recruiting, City of Hope Medical Center | Trial completion date: Oct 2025 --> Sep 2026
    Trial completion date
    |
    HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    sirolimus • clofarabine • melphalan
    5ms
    AALL1131: Combination Chemotherapy in Treating Young Patients With Newly Diagnosed High-Risk B Acute Lymphoblastic Leukemia and Ph-Like TKI Sensitive Mutations (clinicaltrials.gov)
    P3, N=5949, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Oct 2025 --> Oct 2026
    Trial completion date
    |
    ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • ETV6 (ETS Variant Transcription Factor 6)
    |
    MLL rearrangement
    |
    dasatinib • cytarabine • doxorubicin hydrochloride • cyclophosphamide • etoposide IV • methotrexate • vincristine • daunorubicin • clofarabine • leucovorin calcium • Oncaspar liquid (pegaspargase) • mercaptopurine • thioguanine • Hemady (dexamethasone tablets) • Starasid (cytarabine ocfosfate)
    7ms
    VyClo: Vyxeos® With Clofarabine for Pediatric AML (clinicaltrials.gov)
    P1, N=25, Recruiting, Princess Maxima Center for Pediatric Oncology
    New P1 trial
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation) • clofarabine
    7ms
    Treosulfan-Based Versus Clofarabine-Based Conditioning Before Donor Hematopoietic Stem Cell Transplant for the Treatment of Myelodysplastic Syndromes or Acute Myeloid Leukemia (clinicaltrials.gov)
    P2, N=23, Active, not recruiting, Fred Hutchinson Cancer Center | Recruiting --> Active, not recruiting | N=80 --> 23 | Trial completion date: Apr 2027 --> Jul 2026 | Trial primary completion date: Oct 2026 --> Jan 2026
    Enrollment closed • Enrollment change • 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) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-C (Major Histocompatibility Complex, Class I, C)
    |
    clofarabine • fludarabine IV • busulfan • Grafapex (treosulfan)
    7ms
    NCI-2018-01198: Combination Chemotherapy in Treating Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia, Lymphoblastic Lymphoma, Burkitt Lymphoma/Leukemia, or Double-Hit Lymphoma/Leukemia (clinicaltrials.gov)
    P2, N=42, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Aug 2026 --> Aug 2027 | Trial primary completion date: Aug 2025 --> Aug 2027
    Trial completion date • Trial primary completion date
    |
    BCL2 (B-cell CLL/lymphoma 2)
    |
    Rituxan (rituximab) • bortezomib • cyclophosphamide • etoposide IV • clofarabine • Arzerra (ofatumumab) • Truxima (rituximab-abbs) • Marqibo (vincristine liposomal) • Hemady (dexamethasone tablets) • Neulasta (pegfilgrastim) • Neupogen (filgrastim)
    8ms
    Discovery of a novel non-toxic analog of clofarabine for the treatment of triple negative breast cancer. (PubMed, Bioorg Med Chem Lett)
    Our preliminary structure-activity relationship study resulted in the identification of analog 9 with enhanced activity against TNBC cells and improved cytotoxicity. One major advantage of analog 9 is that a pilot dose range finding study demonstrated that this analog elicited no signs of toxicity, whereas clofarabine was not well tolerated.
    Journal • BRCA Biomarker
    |
    BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset)
    |
    clofarabine
    9ms
    Cross-Species Functional Genomic Screens Identify Novel Therapeutic Targets in Malignant Peripheral Nerve Sheath Tumors. (PubMed, Am J Pathol)
    Five druggable targets were selected for validation, with four of the five agents tested proving effective against human MPNST cells (the POLA1 inhibitor clofarabine, DNTT inhibitor cordycepin, BCL6 inhibitor 79-6 and LPAR1/3 inhibitor Ki16425). Clofarabine was especially effective, potently reducing cell numbers at low nanomolar concentrations and inducing a senescent phenotype, possibly via the p53/p21 pathway. These results demonstrate the utility of cross-species functional oncogenomics for the discovery of novel therapeutic targets relevant to human MPNSTs and suggest that clofarabine warrants further evaluation for its therapeutic potential.
    Journal
    |
    BCL6 (B-cell CLL/lymphoma 6) • POLA1 (DNA Polymerase Alpha 1)
    |
    clofarabine • cordycepin (OVI-123)