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    DRUG:

    busulfan

    Company:
    Generic mfg.
    Drug class:
    Alkylating agent
    Related drugs:
    3d
    Proteomic analysis for busulfan-induced spermatogenesis disorder. (PubMed, Ann Med)
    We revealed 190 significantly downregulated proteins in busulfan-treated mouse testes at 30 days and indicated that 70 days is the cut-off point of spermatogenic recovery for busulfan-treated mouse testis, increasing our understanding of this reproductive disorder model. An increased understanding of busulfan's toxic effect will help to prevent and treat reproductive diseases.
    Journal
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    LDHA (Lactate dehydrogenase A) • USP7 (Ubiquitin Specific Peptidase 7)
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    busulfan
    6d
    SAGES1: Gene Editing For Sickle Cell Disease (clinicaltrials.gov)
    P1, N=25, Not yet recruiting, St. Jude Children's Research Hospital | Trial completion date: Dec 2031 --> Dec 2032 | Trial primary completion date: Dec 2028 --> Dec 2029
    Trial completion date • Trial primary completion date
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    CD34 (CD34 molecule)
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    busulfan • plerixafor
    27d
    CD34+ Transplants for Leukemia and Lymphoma (clinicaltrials.gov)
    P2, N=100, Not yet recruiting, Guenther Koehne | Trial completion date: Oct 2031 --> Mar 2032 | Trial primary completion date: Oct 2031 --> Mar 2032
    Trial completion date • Trial primary completion date
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    CD34 (CD34 molecule)
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    cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
    1m
    Human GM-CSF/IL-3 enhance tumor immune infiltration in humanized HCC patient-derived xenografts. (PubMed, bioRxiv)
    NOG (NOD/Shi- scid /IL-2R null ) and NOG-EXL (huGM-CSF/huIL-3 NOG) mice conditioned with Busulfan underwent i.v. injection of human CD34+ cells...In this context, animal models that recapitulate human disease are greatly needed. Leveraging xenograft tumors derived from patients with advanced HCCs and a commercially available immunodeficient mouse strain that expresses human GM-CSF and IL-3, we demonstrate a novel but accessible approach for modeling the HCC tumor microenvironment.
    Journal • PD(L)-1 Biomarker • IO biomarker
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    PD-1 (Programmed cell death 1) • IL2RA (Interleukin 2 receptor, alpha) • CD34 (CD34 molecule) • CSF2 (Colony stimulating factor 2) • IL3 (Interleukin 3)
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    PD-1 expression • CSF2 expression
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    busulfan
    2ms
    BuCy Vs. TBICy for Allo-HSCT in T-ALL Patients (clinicaltrials.gov)
    P3, N=430, Not yet recruiting, The First Affiliated Hospital of Soochow University
    New P3 trial
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    cytarabine • cyclophosphamide • busulfan
    2ms
    Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases (clinicaltrials.gov)
    P2/3, N=38, Completed, Columbia University | Unknown status --> Completed
    Trial completion
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    cyclophosphamide • Campath (alemtuzumab) • fludarabine IV • busulfan
    2ms
    Romidepsin Maintenance After Allogeneic Stem Cell Transplantation (clinicaltrials.gov)
    P1, N=23, Active, not recruiting, Ohio State University Comprehensive Cancer Center | Recruiting --> Active, not recruiting | N=10 --> 23 | Trial completion date: Dec 2022 --> Dec 2024 | Trial primary completion date: Dec 2022 --> Dec 2024
    Enrollment closed • Enrollment change • Trial completion date • Trial primary completion date
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    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
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    Istodax (romidepsin) • fludarabine IV • busulfan
    2ms
    Busulfan Chemotherapy Downregulates TAF7/TNF-α Signaling in Male Germ Cell Dysfunction. (PubMed, Biomedicines)
    Thus, our data indicate a possible function of TAF7 in the regulation of SSCs and spermatogenesis following downregulation by Busulfan. These findings may account for the therapeutic effects of Busulfan and underlie its potential impact on cancer chemotherapy prognosis.
    Journal
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    TNFA (Tumor Necrosis Factor-Alpha)
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    busulfan
    2ms
    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 2026 --> Jul 2027 | Trial primary completion date: Jul 2024 --> Jul 2025
    Trial completion date • Trial primary completion date
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    cyclophosphamide • fludarabine IV • busulfan • Neupogen (filgrastim)
    2ms
    High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly (clinicaltrials.gov)
    P2, N=30, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting
    Enrollment open
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    Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • Promacta (eltrombopag) • busulfan • Nplate (romiplostim)
    2ms
    Evaluation of 3 Different Doses of IV Busulfan (clinicaltrials.gov)
    P2, N=177, Active, not recruiting, Institut Paoli-Calmettes | Unknown status --> Active, not recruiting | Trial completion date: Dec 2018 --> Jul 2026 | Trial primary completion date: Dec 2018 --> Jul 2026
    Enrollment closed • Trial completion date • Trial primary completion date
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    fludarabine IV • busulfan
    3ms
    High-dose Chemotherapy with Thiotepa, Busulfan, and Cyclophosphamide Followed by Autologous Stem Cell Transplantation in Central Nervous System Lymphoma (clinicaltrials.gov)
    P2, N=17, Terminated, Seoul National University Hospital
    New P2 trial
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    cyclophosphamide • thiotepa • busulfan
    3ms
    High activity of the new myeloablative regimen of gemcitabine/clofarabine/busulfan for allogeneic transplant for aggressive lymphomas. (PubMed, Bone Marrow Transplant)
    CD20+ tumors received rituximab. Gem/Clo/Bu patients had better median EFS (12 vs. 3 months, P = 0.001) and OS (25 vs. 7 months, P = 0.003) than 113 Flu/Mel matched-pair controls. The new myeloablative regimen Gem/Clo/Bu has limited toxicity and high activity in allo-SCT for aggressive lymphomas, yielding better outcomes than concurrent matched-pair controls receiving Flu/Mel.
    Journal
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    CD20 (Membrane Spanning 4-Domains A1)
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    gemcitabine • Rituxan (rituximab) • clofarabine • busulfan
    3ms
    NCI-2018-01607: Sorafenib, Busulfan and Fludarabine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia Undergoing Donor Stem Cell Transplant (clinicaltrials.gov)
    P1/2, N=74, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2024 --> Dec 2025 | Trial primary completion date: Dec 2024 --> Dec 2025
    Trial completion date • Trial primary completion date
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    FLT3 positive
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    sorafenib • cyclophosphamide • fludarabine IV • busulfan • Neupogen (filgrastim)
    3ms
    Twice-per-weekSelinexor, 2 Days Melphalan (clinicaltrials.gov)
    P3, N=126, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China
    New P3 trial
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    Xpovio (selinexor) • melphalan • fludarabine IV • busulfan
    3ms
    Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy (clinicaltrials.gov)
    P2, N=22, Active, not recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2024 --> Jun 2025
    Trial completion date
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    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)
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    FLT3-ITD mutation • NPM1 mutation • MLL rearrangement • MLL rearrangement • CEBPA mutation • FLT3 wild-type
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    cyclophosphamide • melphalan • fludarabine IV • busulfan • spanlecortemlocel (MGTA-456)
    3ms
    Olaparib and High-Dose Chemotherapy in Treating Patients With Relapsed or Refractory Lymphomas Undergoing Stem Cell Transplant (clinicaltrials.gov)
    P1, N=50, Active, not recruiting, M.D. Anderson Cancer Center | Trial completion date: Jul 2024 --> Jun 2028 | Trial primary completion date: Jul 2024 --> Jun 2028
    Trial completion date • Trial primary completion date
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    CD34 (CD34 molecule)
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    Lynparza (olaparib) • gemcitabine • Rituxan (rituximab) • Zolinza (vorinostat) • melphalan • Truxima (rituximab-abbs) • busulfan
    3ms
    Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/progenitor Cells in HIV-1 Infected Patients (clinicaltrials.gov)
    P1, N=12, Active, not recruiting, City of Hope Medical Center | Trial completion date: Aug 2024 --> Dec 2024
    Trial completion date
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    CD34 (CD34 molecule) • CD4 (CD4 Molecule)
    |
    busulfan
    3ms
    Thiotepa, fludarabine plus busulfan regimen as conditioning regimen prior to allo-HSCT for adult acute lymphoblastic leukemia:a prospective,single-arm, phase 2 study (ChiCTR2400088533)
    P2, N=80, Not yet recruiting, The First Affiliated Hospital, College of Medicine, Zhejiang University; The First Affiliated Hospital, College of Medicine, Zhejiang University
    New P2 trial
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    fludarabine IV • thiotepa • busulfan
    3ms
    Sendai virus-mediated RNA delivery restores fertility to congenital and chemotherapy-induced infertile female mice. (PubMed, PNAS Nexus)
    This virus also effectively addressed iatrogenic infertility induced by busulfan, a widely used cancer chemotherapy agent. Offspring born through SeV administration and natural mating displayed normal genomic imprinting patterns and fertility. Since SeVs pose no genotoxicity risk, the successful restoration of fertility by SeVs represents a promising approach for treating congenital infertility with somatic cell defects and protecting fertility of cancer patients who may become infertile due to loss of oocytes during cancer therapy.
    Preclinical • Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase)
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    KIT expression
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    busulfan
    4ms
    T-cell large granular lymphocytic leukemia with a STAT3 mutation successfully treated with cord blood transplantation. (PubMed, Intern Med)
    Initial treatment with cyclophosphamide and prednisolone did not improve her condition, but serious infections were observed. The patient underwent cord blood transplantation (CBT) after preconditioning with fludarabine, busulfan, and total body irradiation, yielding a STAT3 Y640F variant disappearance, based on allele-specific quantitative polymerase chain reaction (AS-qPCR). In this case, CBT is a promising refractory T-LGLL treatment option, and the STAT3 Y640F variant AS-qPCR is a T-LGLL activity marker.
    Journal
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    STAT3 (Signal Transducer And Activator Of Transcription 3)
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    cyclophosphamide • fludarabine IV • busulfan
    4ms
    T-cell Large Granular Lymphocytic Leukemia with a STAT3 mutation successfully treated with Cord Blood Transplantation. (PubMed, Intern Med)
    Initial treatment with cyclophosphamide and prednisolone did not improve her condition, but serious infections were observed. The patient underwent cord blood transplantation (CBT) after preconditioning with fludarabine, busulfan, and total body irradiation, yielding a STAT3 Y640F variant disappearance, based on allele-specific quantitative polymerase chain reaction (AS-qPCR). In this case, CBT is a promising refractory T-LGLL treatment option, and the STAT3 Y640F variant AS-qPCR is a T-LGLL activity marker.
    Journal
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    STAT3 (Signal Transducer And Activator Of Transcription 3)
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    cyclophosphamide • fludarabine IV • busulfan
    4ms
    Uproleselan With Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for AML (clinicaltrials.gov)
    P1, N=1, Terminated, Malika Kapadia | Phase classification: P1/2 --> P1 | N=28 --> 1 | Trial completion date: Dec 2028 --> Dec 2023 | Recruiting --> Terminated | Trial primary completion date: Dec 2027 --> Oct 2023; On 7/26/24 the Sponsor-Investigator was notified GlycoMimetics was terminating contracting for NCT05569512 following company restructuring. This notification came ahead of the study meeting criteria to progress from Phase 1 to Phase 2.
    Phase classification • Enrollment change • Trial completion date • Trial termination • Trial primary completion date • Pre-transplantation
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    RUNX1 (RUNX Family Transcription Factor 1) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
    |
    clofarabine • fludarabine IV • busulfan • uproleselan sodium (APL-106)
    4ms
    A Prospective, Open-label, Randomized Controlled, Multicenter Clinical Study of TBI-containing or TMLI-containing Pretreatment Regimen for Adult Acute Lymphocytic Sibling Alloidentical Hematopoietic Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=170, Not yet recruiting, The First Affiliated Hospital of Zhengzhou University
    New P2 trial
    |
    busulfan
    4ms
    A Single-Arm, Prospective Study of TBI+BUMEL as Conditioning for SCT2 in Patients With Malignant Hematologic Diseases (clinicaltrials.gov)
    P=N/A, N=40, Recruiting, The First Affiliated Hospital of Soochow University
    New trial
    |
    melphalan • busulfan
    4ms
    Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (clinicaltrials.gov)
    P1/2, N=85, Recruiting, Essen Biotech | Not yet recruiting --> Recruiting | Initiation date: Nov 2024 --> Aug 2024
    Enrollment open • Trial initiation date • Gene therapy • Viral vector
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    busulfan
    5ms
    Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study (clinicaltrials.gov)
    P4, N=823, Completed, First Affiliated Hospital of Guangxi Medical University | Recruiting --> Completed | Trial completion date: May 2023 --> Oct 2023
    Trial completion • Trial completion date
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    cyclophosphamide • fludarabine IV • busulfan
    5ms
    ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene (clinicaltrials.gov)
    P1, N=10, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Jul 2024 --> Jul 2025 | Trial primary completion date: Jul 2024 --> Jul 2025
    Trial completion date • Trial primary completion date • Viral vector
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    CD34 (CD34 molecule)
    |
    busulfan • Neupogen (filgrastim)
    5ms
    Pre-transplant Purging and Post-transplant MRD-guided Maintenance Therapy With Elranatamab in Patients With High-risk Multiple Myeloma (clinicaltrials.gov)
    P2, N=40, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting
    Enrollment open • Pre-transplantation • Post-transplantation
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    lenalidomide • Elrexfio (elranatamab-bcmm) • busulfan • captisol-enabled melphalan • plerixafor
    5ms
    Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes (clinicaltrials.gov)
    P2, N=38, Active, not recruiting, University of Illinois at Chicago | Recruiting --> Active, not recruiting | Trial completion date: Apr 2024 --> Apr 2026 | Trial primary completion date: Apr 2024 --> Apr 2026
    Enrollment closed • Trial completion date • Trial primary completion date
    |
    FLT3 (Fms-related tyrosine kinase 3) • KMT2A (Lysine Methyltransferase 2A) • 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)
    |
    methotrexate • fludarabine IV • busulfan
    5ms
    CY-MET-RIC: Study Testing Two Conditioning Regimen With a Single Prophylaxis of GVHD by Cyclophosphamide and Methotrexate Post-transplant in Patients Eligible for Matched-donor Allograft Transplantation (clinicaltrials.gov)
    P2, N=82, Recruiting, Nantes University Hospital | Not yet recruiting --> Recruiting
    Enrollment open • Post-transplantation
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    cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan
    5ms
    Intensive Conditioning withTHI/Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement (clinicaltrials.gov)
    P=N/A, N=50, Recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Not yet recruiting --> Recruiting
    Enrollment open
    |
    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)
    |
    cytarabine • fludarabine IV • thiotepa • busulfan
    5ms
    Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) (clinicaltrials.gov)
    P=N/A, N=30, Recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Jun 2024 --> Jun 2025 | Trial primary completion date: Jun 2024 --> Jun 2025
    Trial completion date • Trial primary completion date
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    fludarabine IV • busulfan
    5ms
    Gene Editing For Sickle Cell Disease (clinicaltrials.gov)
    P1, N=25, Not yet recruiting, St. Jude Children's Research Hospital
    New P1 trial
    |
    CD34 (CD34 molecule)
    |
    busulfan • plerixafor
    5ms
    An International, Multicenter, Phase I Open Label Study to Evaluate Safety and Efficacy of Administration of Autologous CD34+ Cells Gene Edited Ex Vivo Using a CRISPR/AAV6 Platform to Introduce a Codon Optimized cDNA Version of the Red-Cell Type Pyruvate Kinase Genein Adult and Pediatric Patients with Severe Pyruvate Kinase (ChiCTR2300073795)
    P=N/A, N=2, Recruiting, Shanghai Children's Medical Center; DanausGT biotechnology Co.,Ltd. | Not yet recruiting --> Recruiting | Initiation date: Jul 2023 --> Nov 2023
    Enrollment open • Trial initiation date
    |
    busulfan
    5ms
    Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma (clinicaltrials.gov)
    P1, N=150, Completed, Children's Oncology Group | Active, not recruiting --> Completed
    Trial completion • Trial completion date
    |
    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
    |
    cisplatin • doxorubicin hydrochloride • cyclophosphamide • etoposide IV • vincristine • topotecan • melphalan • busulfan • Neupogen (filgrastim)
    6ms
    A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies (clinicaltrials.gov)
    P2, N=3, Terminated, St. Jude Children's Research Hospital | N=32 --> 3 | Trial completion date: Apr 2027 --> May 2024 | Recruiting --> Terminated | Trial primary completion date: Apr 2026 --> May 2024; Principal Investigator left institution
    Enrollment change • Trial completion date • Trial termination • Trial primary completion date
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    Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • mesna • thiotepa • busulfan • cyclosporine
    6ms
    CD34+ Transplants for Leukemia and Lymphoma (clinicaltrials.gov)
    P2, N=100, Not yet recruiting, Guenther Koehne | Trial completion date: Jun 2031 --> Oct 2031 | Trial primary completion date: Jun 2031 --> Oct 2031
    Trial completion date • Trial primary completion date
    |
    CD34 (CD34 molecule)
    |
    cyclophosphamide • melphalan • fludarabine IV • thiotepa • busulfan
    6ms
    Giving Chemotherapy for a Shortened Amount of Time Before a Stem Cell Transplantation (clinicaltrials.gov)
    P1, N=39, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    TP53 (Tumor protein P53) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
    |
    melphalan • fludarabine IV • busulfan
    6ms
    Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia (clinicaltrials.gov)
    P2, N=33, Active, not recruiting, Masonic Cancer Center, University of Minnesota | Recruiting --> Active, not recruiting | N=20 --> 33 | Trial primary completion date: Dec 2025 --> Jun 2024
    Enrollment closed • Enrollment change • Trial primary completion date
    |
    KMT2A (Lysine Methyltransferase 2A)
    |
    melphalan • fludarabine IV • busulfan • Neupogen (filgrastim)
    6ms
    Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS (clinicaltrials.gov)
    P2, N=324, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2024 --> Dec 2027 | Trial primary completion date: Dec 2024 --> Dec 2027
    Trial completion date • Trial primary completion date
    |
    KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • DNMT3A (DNA methyltransferase 1) • NF1 (Neurofibromin 1) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • PTPN1 (Protein Tyrosine Phosphatase Non-Receptor Type 1)
    |
    Venclexta (venetoclax) • cladribine • fludarabine IV • thiotepa • busulfan
    6ms
    CD34 Selected Allogeneic HCT w/ Myeloablative Conditioning Plus CD8+ Memory TCell Infusion in MDS, AL and CML (clinicaltrials.gov)
    P2, N=7, Completed, Stanford University | Suspended --> Completed | N=20 --> 7 | Trial completion date: Feb 2026 --> Sep 2023 | Trial primary completion date: Feb 2025 --> Sep 2023
    Trial completion • Enrollment change • Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1)
    |
    cyclophosphamide • fludarabine IV • thiotepa • busulfan