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    CANCER:

    Myelodysplastic Syndrome

    16h
    VYSION: VYxeoS Liposomal Italian Observational Study iN the Real Practice (clinicaltrials.gov)
    P=N/A, N=112, Active, not recruiting, Jazz Pharmaceuticals | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation)
    1d
    DNA and RNA NGS for Myeloid Neoplasms Using Oncomine Myeloid Assay GX v2 on GeneXus: An Assessment of Clinical Utility (AMP 2024)
    This DNA- and RNA-based 80-gene panel has proven to be a powerful tool for genomic profiling of myeloid neoplasms. The results were provided to hematopathologists/oncologists in timely fashion with the critical information for diagnosis confirmation, and disease classification, as well as assessment of patient response to treatment.
    Clinical • Next-generation sequencing
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    KRAS (KRAS proto-oncogene GTPase) • TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • NRAS (Neuroblastoma RAS viral oncogene homolog) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • NPM1 (Nucleophosmin 1) • DNMT3A (DNA methyltransferase 1) • JAK2 (Janus kinase 2) • RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • TET2 (Tet Methylcytosine Dioxygenase 2) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • STAG2 (Stromal Antigen 2) • DDX41 (DEAD-Box Helicase 41) • CALR (Calreticulin) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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    FLT3-ITD mutation • NPM1 mutation • U2AF1 mutation • CEBPA mutation • JAK2 V617F
    |
    Oncomine Myeloid Assay GX
    2d
    NCI-2019-03057: Using the Anticancer Drug Olaparib to Treat Relapsed/Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome With an Isocitrate Dehydrogenase (IDH) Mutation (clinicaltrials.gov)
    P2, N=14, Active, not recruiting, National Cancer Institute (NCI) | N=94 --> 14 | Trial completion date: Dec 2024 --> Nov 2025 | Trial primary completion date: Dec 2024 --> Jan 2024
    Enrollment change • Trial completion date • Trial primary completion date
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
    |
    IDH2 mutation • IDH1 R132H • IDH2 R172K • IDH1 R132C • IDH wild-type • IDH1 R132 • IDH1 R132G • IDH2 R140Q • IDH1 R132L • IDH1 R132S • IDH1 R132V • IDH2 R172 • IDH2 R172G
    |
    Lynparza (olaparib)
    2d
    A Safety, Tolerability and Efficacy Study of NC525 in Subjects with Advanced Myeloid Neoplasms (clinicaltrials.gov)
    P1, N=63, Active, not recruiting, NextCure, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    NC525
    3d
    ODYSSEY: First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients (clinicaltrials.gov)
    P1/2, N=36, Recruiting, Advanced BioDesign | Phase classification: P1 --> P1/2
    Phase classification
    4d
    Efficacy of HLA epitope-matched platelet transfusion (ChiCTR2400091253)
    P=N/A, N=700, Not yet recruiting, Qilu Hospital of Shandong University; Qilu Hospital of Shandong University
    New trial
    4d
    Efficacy of decitabine in CR1 stage of AML/intermediate and high-risk MDS allogeneic transplantation conditioning regimens (ChiCTR2400091268)
    P=N/A, N=76, Recruiting, Ningbo First Hospital; Ningbo First Hospital
    New trial
    |
    decitabine
    4d
    Study of an application-delivered mindfulness-based intervention to manage fear of progression for patients with hematologic malignancies (ChiCTR2400090963)
    P=N/A, N=90, Completed, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; Institute of Hematology
    New trial
    4d
    A novel myeloablative pretreatment scheme for single arm, single center, exploratory study of umbilical cord blood transplantation (ChiCTR2400090099)
    P=N/A, N=30, Not yet recruiting, The Seventh Affiliated Hospital Sun Yat-sen University; The?Seventh?Affiliated?Hospital?Sun?Yat-sen?University
    New trial
    |
    cyclophosphamide • fludarabine IV
    4d
    Phase II clinical study on the efficacy, safety, and pharmacokinetics of HS-20106 in IPSS-R extremely low-risk, low-risk, or moderate risk myelodysplastic syndrome (MDS) anemia subjects (ChiCTR2400090140)
    P2, N=176, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.; Institute of Hematolog
    New P2 trial
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    elritercept (KER-050)
    7d
    LANA: Creation of a Biocollection of Patients with Acute Myeloid Leukemia (AML) or Lymphoid Leukemia (ALL) or High-risk Myelodysplastic Syndrome (MDS) Monitored At the Nantes University Hospital (clinicaltrials.gov)
    P=N/A, N=1000, Not yet recruiting, Nantes University Hospital
    New trial
    7d
    MCC-20963: Fedratinib in Myelodysplastic /Myeloproliferative Neoplasms (MDS/MPNs) and Chronic Neutrophilic Leukemia (CNL) (clinicaltrials.gov)
    P2, N=25, Active, not recruiting, H. Lee Moffitt Cancer Center and Research Institute | Recruiting --> Active, not recruiting
    Enrollment closed
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    ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase)
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    Inrebic (fedratinib)
    7d
    8-Chloroadenosine in Combination With Venetoclax for the Treatment of Patients With Relapsed/Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=30, Suspended, City of Hope Medical Center | Recruiting --> Suspended
    Trial suspension • Combination therapy
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    Venclexta (venetoclax)
    8d
    5-Azacitidine and Decitabine Epigenetic Therapy for Myeloid Malignancies (clinicaltrials.gov)
    P1, N=20, Recruiting, Benjamin Tomlinson | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
    Trial completion date • Trial primary completion date
    |
    ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • CD14 (CD14 Molecule) • DCK (Deoxycytidine Kinase 2) • DNMT1 (DNA methyltransferase 1) • ITGAM (Integrin, alpha M) • NT5C (5', 3'-Nucleotidase, Cytosolic)
    |
    Chr del(5q)
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    azacitidine • decitabine
    8d
    Genetic abnormalities in bone marrow failure (PubMed, Rinsho Ketsueki)
    Notably, the presence of PNH-type cells and HLA class I allele-lacking cells represent two major instances of escape hematopoiesis, which indicate the presence of HSCs evading autoimmune T cell attacks. These findings provide crucial insights into the immune pathophysiology of BM failure.
    Journal
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    DNMT3A (DNA methyltransferase 1) • ASXL1 (ASXL Transcriptional Regulator 1) • BCOR (BCL6 Corepressor) • BCORL1 (BCL6 Corepressor Like 1)
    8d
    Validation of the Revised 2022 European LeukemiaNet Risk Stratification in Adult Patients with Acute Myeloid Leukemia. (PubMed, Blood Adv)
    We aimed to validate the prognostic value of the 2022 ELN classification (ELN22) evaluating 1,570 newly diagnosed AML patients (median age, 56 years) treated with cytarabine-based intensive chemotherapy regimens...In conclusion, the ELN22 risk stratification improves prognostic discrimination in a large cohort of intensively treated AML patients. Given the heterogeneous outcome in patients with MR gene alterations, ranging between those of intermediate and adverse risk patients, we suggest reevaluation of risk allocation in these patients.
    Journal
    |
    TP53 (Tumor protein P53) • EZH2 (Enhancer of zeste 2 polycomb repressive complex 2 subunit) • STAG2 (Stromal Antigen 2) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
    |
    TP53 mutation • EZH2 mutation • STAG2 mutation • ZRSR2 mutation
    |
    cytarabine
    8d
    BoHemE: Study Investigating the Interactions of Bone and Hematopoiesis in the Elderly (clinicaltrials.gov)
    P=N/A, N=297, Completed, Technische Universität Dresden | Recruiting --> Completed | Trial completion date: Dec 2023 --> Aug 2024 | Trial primary completion date: Dec 2023 --> Aug 2024
    Trial completion • Trial completion date • Trial primary completion date
    8d
    Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors (clinicaltrials.gov)
    P1, N=22, Recruiting, University of Wisconsin, Madison | Suspended --> Recruiting | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
    Enrollment open • Trial completion date • Trial primary completion date
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    melphalan • zoledronic acid
    8d
    PEVENAZA: A Study of Pevonedistat and Venetoclax Combined With Azacitidine to Treat Acute Myeloid Leukemia (AML) in Adults Unable to Receive Intensive Chemotherapy (clinicaltrials.gov)
    P2, N=164, Active, not recruiting, Takeda | Trial completion date: Sep 2024 --> Dec 2024
    Trial completion date
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    Venclexta (venetoclax) • azacitidine • pevonedistat (MLN4924)
    9d
    Orca-T Following Chemotherapy and Total Marrow and Lymphoid Irradiation for the Treatment of Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia or Myelodysplastic Syndrome (clinicaltrials.gov)
    P1, N=33, Recruiting, City of Hope Medical Center | Initiation date: Sep 2024 --> May 2024
    Trial initiation date
    |
    melphalan • fludarabine IV • thiotepa • Orca-T
    9d
    Study of Standard Intensive Chemotherapy Versus Intensive Chemotherapy with CPX-351 in Adult Patients with Newly Diagnosed AML and Intermediate- or Adverse Genetics (clinicaltrials.gov)
    P3, N=882, Recruiting, University of Ulm | Trial completion date: Mar 2024 --> Jun 2027 | Trial primary completion date: Mar 2024 --> Jun 2027
    Trial completion date • Trial primary completion date
    |
    Vyxeos (cytarabine/daunorubicin liposomal formulation)
    9d
    Beneficial effects of cellular immunotherapy in the prevention and treatment of posttransplant hematologic relapse of myelodysplastic neoplasms. (PubMed, Ann Hematol)
    In HemRel, using cIMTx demonstrated a significantly superior OS rate compared to non-cIMTx modalities (25.8% vs. 6.1% vs. 0%, P < .001). In summary, cIMTx demonstrated superior outcomes in both pre-HemRel and HemRel groups, proving particularly advantageous in pre-HemRel cases with progression risk factors, while its benefits remained consistent in HemRel cases.
    Journal • Post-transplantation
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    WT1 (WT1 Transcription Factor)
    9d
    TP53 Mutation Status in Myelodysplastic Neoplasm and Acute Myeloid Leukemia: Impact of Reclassification Based on the 5th WHO and International Consensus Classification Criteria: A Korean Multicenter Study. (PubMed, Ann Lab Med)
    According to the 5th WHO classification and ICC, the multi-hit TP53 mutation criteria were met in 58.6% and 75% of MDS patients, respectively, and the primary determinants were a TP53 VAF >50% for the 5th WHO classification and the presence of a complex karyotype for the ICC. Collectively, we elucidated the molecular genetic characteristics of patients with TP53-mutated MDS and AML, highlighting key factors in applying TP53 mutation-related criteria in updated classifications, which will aid in establishing diagnostic strategies.
    Clinical • Journal
    |
    TP53 (Tumor protein P53)
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    TP53 mutation • MET mutation
    9d
    HM2023-05: GTB-3650 Trike for High Risk MDS and R/R AML (clinicaltrials.gov)
    P1, N=45, Not yet recruiting, Masonic Cancer Center, University of Minnesota | Initiation date: Oct 2024 --> Jan 2025
    Trial initiation date • Trispecific
    |
    GTB-3650
    10d
    Inducible Pluripotent Stem Cell Models to Study Bone Marrow Failure and MDS Predisposition Syndromes. (PubMed, Exp Hematol)
    The review also explores future directions, including the potential of iPSC models for drug discovery and personalized medicine approaches. This review underscores the significance of iPSC technology in advancing our understanding of inherited hematopoietic disorders and its potential to inform novel therapeutic strategies.
    Review • Journal
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    RUNX1 (RUNX Family Transcription Factor 1) • ETV6 (ETS Variant Transcription Factor 6) • ALDH2 (Aldehyde Dehydrogenase 2 Family Member) • GATA2 (GATA Binding Protein 2) • ANKRD26 (Ankyrin Repeat Domain Containing 26)
    10d
    Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for Hematological Diseases (clinicaltrials.gov)
    P=N/A, N=200, Recruiting, Masonic Cancer Center, University of Minnesota | Trial completion date: Oct 2025 --> Oct 2026 | Trial primary completion date: Oct 2024 --> Oct 2025
    Trial completion date • Trial primary completion date
    |
    KMT2A (Lysine Methyltransferase 2A) • IKZF1 (IKAROS Family Zinc Finger 1)
    |
    MLL rearrangement • MLL rearrangement
    |
    cyclophosphamide • fludarabine IV • cyclosporin A microemulsion
    10d
    KT253-AL-101: Safety and Clinical Activity of KT-253 in Adult Patients with High Grade Myeloid Malignancies, Acute Lymphocytic Leukemia, Lymphoma, Solid Tumors (clinicaltrials.gov)
    P1, N=70, Active, not recruiting, Kymera Therapeutics, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed • Metastases
    11d
    Pevonedistat and Azacitidine in MDS or MDS/MPN Patients Who Fail Primary Therapy With DNA Methyl Transferase Inhibitors (clinicaltrials.gov)
    P2, N=71, Completed, Vanderbilt-Ingram Cancer Center | Active, not recruiting --> Completed | Trial completion date: Oct 2024 --> Nov 2023
    Trial completion • Trial completion date • Tumor mutational burden
    |
    azacitidine • pevonedistat (MLN4924)
    11d
    Uproleselan, Cladribine, and Low Dose Cytarabine for the Treatment of Patients With Treated Secondary Acute Myeloid Leukemia (clinicaltrials.gov)
    P1/2, N=37, Completed, M.D. Anderson Cancer Center | Active, not recruiting --> Completed | Trial completion date: Dec 2026 --> Oct 2024 | Trial primary completion date: Dec 2026 --> Oct 2024
    Trial completion • Trial completion date • Trial primary completion date
    |
    cytarabine • cladribine • uproleselan sodium (APL-106) • Starasid (cytarabine ocfosfate)
    13d
    Single-cell analysis of bone marrow CD8+ T cells in Myeloid Neoplasms reveals pathways associated with disease progression and response to treatment with Azacitidine. (PubMed, Cancer Res Commun)
    The response to AZA was positively associated with the enrichment of IFN-mediated pathways, whereas an enhanced TGF-β signaling signature was observed in non-responders. Our results suggest that targeting CD8+ T cells with inhibitors of TGF-β signaling in combination with AZA is a potential therapeutic strategy for HR-MDS and AML.
    Journal
    |
    CD8 (cluster of differentiation 8) • TGFB1 (Transforming Growth Factor Beta 1) • CXCR3 (C-X-C Motif Chemokine Receptor 3) • B3GAT1 (Beta-1,3-Glucuronyltransferase 1)
    |
    azacitidine
    13d
    Phase I First-in-Human Dose Escalation Study of the oral Casein Kinase 1α and Cyclin Dependent Kinase 7/9 inhibitor BTX-A51 in advanced MDS and AML. (PubMed, Res Sq)
    Ex-vivo studies confirmed higher efficacy of BTX-A51 on RUNX1 -mutated myeloblasts and demonstrate synergy with azacitidine and venetoclax. Although the overall efficacy was modest, this study lays the groundwork for future studies with improved patient selection and combination approaches.
    P1 data • Journal • Metastases
    |
    RUNX1 (RUNX Family Transcription Factor 1) • CDK7 (Cyclin Dependent Kinase 7)
    |
    RUNX1 mutation • MCL1 expression • TP53 expression
    |
    Venclexta (venetoclax) • azacitidine • BTX-A51
    14d
    Trial of 2 Step ATG for Acute GVHD Prevention Post Myeloablative Allogeneic Stem Cell Transplant (clinicaltrials.gov)
    P2, N=29, Not yet recruiting, University of Alabama at Birmingham | Initiation date: Jun 2024 --> Nov 2024 | Trial primary completion date: Oct 2025 --> Jan 2026
    Trial initiation date • Trial primary completion date
    |
    cyclophosphamide
    14d
    TAK-243 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes With Increased Blasts (clinicaltrials.gov)
    P1, N=42, Recruiting, National Cancer Institute (NCI) | Not yet recruiting --> Recruiting | N=72 --> 42 | Trial completion date: Dec 2024 --> Oct 2026 | Trial primary completion date: Dec 2024 --> Oct 2026
    Enrollment open • Enrollment change • Trial completion date • Trial primary completion date
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    CD4 (CD4 Molecule)
    |
    TAK-243
    14d
    Oral Arsenic (ATO) in Low-risk Myelodysplastic Syndromes (MDS) (clinicaltrials.gov)
    P1, N=24, Not yet recruiting, Groupe Francophone des Myelodysplasies
    New P1 trial
    |
    arsenic trioxide • Reblozyl (luspatercept-aamt)
    14d
    A Study of ASTX030 (Cedazuridine in Combination With Azacitidine) in MDS, CMML, or AML (clinicaltrials.gov)
    P2/3, N=317, Active, not recruiting, Taiho Oncology, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed • Combination therapy
    |
    cedazuridine/azacitidine (ASTX030)
    14d
    Diagnosis and Risk Stratification of Acute Myeloid Leukemia, Myelodysplasia -Related (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi)
    Molecular genetic information plays a crucial role in diagnosing AML-MR, highlighting the importance of genetics in diagnosis and prognosis. Most AML-MR patients fall into poor prognosis categories, necessitating early intensive and targeted therapy for better survival outcomes.
    Journal
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    ASXL1 (ASXL Transcriptional Regulator 1) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor)
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    ASXL1 mutation • SRSF2 mutation • BCOR mutation
    14d
    Clinical Analysis of CD4+CD8- T-Cell Large Granular Lymphocytic[JP] Leukemia (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi)
    CD4+CD8- T-LGLL is very rare in clinical practice, and its clinical manifestations are different from those of CD4-CD8+ T-LGLL.
    Journal
    |
    CD8 (cluster of differentiation 8) • CD4 (CD4 Molecule)
    |
    cyclophosphamide • prednisone
    14d
    Interferon-γ (IFN-γ) With Donor Leukocyte Infusion to Treat Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes Post Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=45, Recruiting, Sawa Ito, MD | Trial primary completion date: Aug 2026 --> Oct 2027
    Trial primary completion date • Combination therapy
    |
    IFNG (Interferon, gamma)
    |
    Actimmune (interferon gamma-1 b)
    14d
    FLAER Revealed Normally Expected Non-PNH FLAER-Dim Immature Myeloid Cells (CD117+/CD34-) In Bone Marrow Aspirates and Could Be Utilized as a Marker of Hierarchical Hematopoiesis. (PubMed, Int J Lab Hematol)
    The application of FLAER in PNH-positive and PNH-negative reactive or malignant BM aspirates identified normally expected non-PNH FLAER-dim CD34-/CD117+/HLA-DR+/CD33+ myeloid precursors in all samples. A specific FLAER-associated maturation pattern was observed, which is proposed for further study within MRD and diagnostic protocols.
    Journal • IO biomarker
    |
    KIT (KIT proto-oncogene, receptor tyrosine kinase) • CD38 (CD38 Molecule) • CD123 (Interleukin 3 Receptor Subunit Alpha) • CD33 (CD33 Molecule) • CD34 (CD34 molecule) • PTPRC (Protein Tyrosine Phosphatase Receptor Type C) • CD14 (CD14 Molecule) • CEACAM6 (CEA Cell Adhesion Molecule 6) • IL3RA (Interleukin 3 Receptor Subunit Alpha) • CEACAM8 (CEA Cell Adhesion Molecule 8)
    15d
    A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. (PubMed, Expert Rev Hematol)
    Olutasidenib, enasidenib, and ivosidenib are approved for relapsed AML...Ivosidenib + azacitidine demonstrated a survival benefit not seen with enasidenib + azacitidine. It is unclear whether newly diagnosed AML should be treated with azacitidine + ivosidenib or azacitidine + venetoclax...Single arm studies show post-transplant maintenance is safe, however, randomized trials are needed. Similarly, IDH inhibitors can be combined with chemotherapy however randomized studies are needed.
    Review • Journal
    |
    IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
    |
    Venclexta (venetoclax) • azacitidine • Tibsovo (ivosidenib) • Idhifa (enasidenib) • Rezlidhia (olutasidenib)
    15d
    Triplex Vaccine in Preventing CMV Infection in Patients Undergoing Hematopoietic Stem Cell Transplantation (clinicaltrials.gov)
    P2, N=7, Active, not recruiting, City of Hope Medical Center | N=12 --> 7
    Enrollment change • Triplex vaccine
    |
    Triplex (CMV-MVA vaccine)
    16d
    Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia (clinicaltrials.gov)
    P1/2, N=52, Recruiting, Uma Borate | Not yet recruiting --> Recruiting
    Enrollment open • Metastases
    |
    azacitidine • Niktimvo (axatilimab-csfr)