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Congenital Bone Marrow Failure Syndromes

NCT06839456 2026-04-15

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Classical hematologyLymphoidOther hematologic neoplasm

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Anemia, Refractory, with Excess of Blasts, Hemoglobinopathies, Immunologic Deficiency Syndromes, Leukemia, Lymphoma, Non-Hodgkin, Metabolism, Inborn Errors

NCT05796570 2026-03-19

Phase 2 Recruiting

37 enrolled

Classical hematologyMyeloidOther hematologic neoplasm

Congenital Bone Marrow Failure Syndromes, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

NCT02958462 2026-02-23

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Phase NA Recruiting

2,000 enrolled

Classical hematologyOther hematologic neoplasm

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Cytopenia, Neoplastic Syndromes, Hereditary, Hematologic Neoplasms

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Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

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Pre-myeloid Cancer and Bone Marrow Failure Clinic Study