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    CANCER:

    Myelodysplastic Syndrome

    1m
    CASPALLO: Allodepleted T Cells Transduced With Inducible Caspase 9 Suicide Gene (clinicaltrials.gov)
    P1, N=10, Active, not recruiting, Baylor College of Medicine | Trial completion date: Jul 2026 --> Jul 2027
    Trial completion date
    1m
    A Vaccine (CMV-MVA Triplex Vaccine) for the Enhancement of CMV-Specific Immunity and the Prevention of CMV Viremia in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplant (clinicaltrials.gov)
    P1, N=46, Recruiting, City of Hope Medical Center | Trial completion date: Feb 2030 --> May 2028 | Trial primary completion date: Feb 2030 --> May 2028
    Trial completion date • Trial primary completion date
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    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-DPB1 (Major Histocompatibility Complex, Class II, DP Beta 1) • HLA-C (Major Histocompatibility Complex, Class I, C)
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    Triplex (CMV-MVA vaccine)
    1m
    Thiotepa-based Conditioning Regimen With De-escalated Post-graft Cyclophosphamide for Allogeneic Stem Cell Transplantation in Hematologic Malignancies (clinicaltrials.gov)
    P1, N=48, Recruiting, Sawa Ito, MD | Not yet recruiting --> Recruiting
    Enrollment open
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    BCL2 (B-cell CLL/lymphoma 2) • 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)
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    cyclophosphamide • fludarabine IV • thiotepa • busulfan
    1m
    A Novel GATA2 Splice-Site Mutation Associated with Familial Myelodysplastic Syndrome. (PubMed, Pediatr Allergy Immunol Pulmonol)
    Genetic analysis identified a previously unreported heterozygous splice-site variant in GATA2 (c.1017 + 1 G > A), confirmed in stored DNA from her deceased father. Despite supportive care and allogeneic hematopoietic stem cell transplantation from a matched unrelated donor, she developed severe graft-versus-host disease and died from transplant-related complications.This report identifies a previously unreported GATA2 splice-site variant with clinical and familial evidence supporting pathogenicity, contributing to the expanding mutational spectrum and enhancing understanding of genotype-phenotype correlations in GATA2 deficiency.
    Journal
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    GATA2 (GATA Binding Protein 2)
    1m
    Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes (clinicaltrials.gov)
    P2, N=49, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Apr 2026 --> Apr 2027
    Trial completion date
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    RUNX1 (RUNX Family Transcription Factor 1) • SF3B1 (Splicing Factor 3b Subunit 1) • ASXL1 (ASXL Transcriptional Regulator 1) • SRSF2 (Serine and arginine rich splicing factor 2) • BCOR (BCL6 Corepressor) • U2AF1 (U2 Small Nuclear RNA Auxiliary Factor 1) • STAG2 (Stromal Antigen 2) • ZRSR2 (Zinc Finger CCCH-Type, RNA Binding Motif And Serine/Arginine Rich 2)
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    Chr del(11q)
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    Vyxeos (cytarabine/daunorubicin liposomal formulation) • pomalidomide
    1m
    CH CARE: Clonal Hematopoiesis Chemotherapy and Radiation Effects Study (clinicaltrials.gov)
    P=N/A, N=5000, Recruiting, Dana-Farber Cancer Institute
    New trial
    1m
    Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT (clinicaltrials.gov)
    P2, N=120, Not yet recruiting, Peking University People's Hospital
    New P2 trial
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    cytarabine • cyclophosphamide • fludarabine IV • busulfan
    1m
    ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions (clinicaltrials.gov)
    P3, N=402, Active, not recruiting, Bristol-Myers Squibb | Trial completion date: Mar 2030 --> May 2031 | Trial primary completion date: Jun 2027 --> Mar 2028
    Trial completion date • Trial primary completion date
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    Reblozyl (luspatercept-aamt)
    1m
    Phase I-II PTCy, Bortezomib and Sitagliptin for Prevention of GvHD Following Allogeneic HSCT (clinicaltrials.gov)
    P1/2, N=72, Not yet recruiting, Northwell Health
    New P1/2 trial
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    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)
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    bortezomib • cyclophosphamide • Orencia (abatacept)
    1m
    Bloom Syndrome Presenting With Early-Onset Myelodysplastic Syndrome and Triple Overlapping Vascular Neurocutaneous Phenotypes: A Case Report. (PubMed, Clin Case Rep)
    The course was complicated by severe sepsis, leading to death before hematopoietic stem cell transplantation could be performed. This case highlights the importance of considering inherited cancer predisposition syndromes in children with cytopenia, growth failure, and complex neurocutaneous phenotypes.
    Journal
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    BLM (BLM RecQ Like Helicase)
    1m
    ALICE: Iadademstat in Combination With Azacitidine and Venetoclax in Treating Newly Diagnosed Acute Myeloid Leukemia (clinicaltrials.gov)
    P1, N=30, Recruiting, OHSU Knight Cancer Institute | Trial completion date: May 2026 --> May 2028 | Trial primary completion date: Mar 2026 --> Mar 2027
    Trial completion date • Trial primary completion date
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    ABL1 (ABL proto-oncogene 1)
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    ABL1 fusion
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    Venclexta (venetoclax) • azacitidine • iadademstat (ORY-1001)
    1m
    CD64 CAR T Cell Therapy in Adults With Relapsed and/or Refractory AML (clinicaltrials.gov)
    P1, N=23, Recruiting, University of Colorado, Denver | Not yet recruiting --> Recruiting
    Enrollment open