Congenital Neutropenia

• CLINICAL_TRIALS trial_result · 2026-03-12
  Clinical Trial Update: Screening for Genes in Patients With Congenital Neutropenia

  This clinical trial update describes a completed study evaluating gene screening in patients with congenital neutropenia to identify genetic…

• CLINICAL_TRIALS research · 2025-11-04
  Clinical Trial Update: Investigation of the Genetics of Hematologic Diseases

  A St. Jude-sponsored clinical trial update indicates a study on the genetics of hematologic diseases is currently recruiting, covering sever…

• CLINICAL_TRIALS trial_result · 2025-08-06
  Clinical Trial Update: A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders [Phase 1, Phase 2]

  A completed Phase 1/Phase 2 clinical trial evaluating mavorixafor in participants with congenital neutropenia and chronic idiopathic neutrop…

• CLINICAL_TRIALS research · 2025-07-15
  Clinical Trial Update: Serial Blood Count Study [EARLY_Phase 1]

  An early-phase 1 serial blood count study investigating neutropenia is currently recruiting at the University of Washington. The trial focus…

• CLINICAL_TRIALS research · 2025-05-31
  Clinical Trial Update: Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform

  Describes a global Shwachman-Diamond Syndrome patient survey and partnering platform sponsored by the Shwachman-Diamond Syndrome Alliance, I…

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• CLINICAL_TRIALS trial_result · 2025-03-03
  Clinical Trial Update: Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies [NA]

  A hematopoietic stem cell transplant trial for high-risk hemoglobinopathies has been terminated; the conditions studied include sickle cell …

• CLINICAL_TRIALS trial_result · 2015-03-09
  Clinical Trial Update: Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation [NA]

  Clinical trial update indicating termination of an Alefacept plus allogeneic hematopoietic stem cell transplantation study, with results pos…

• CLINICAL_TRIALS trial_result · 2013-05-16
  Clinical Trial Update: Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders [Phase 2]

  Phase 2 trial evaluating a conditioning regimen of alemtuzumab, fludarabine, and busulfan followed by donor stem cell transplant in young pa…

• CLINICAL_TRIALS trial_result · 2012-11-12
  Clinical Trial Update: Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission [Phase 1, Phase 2]

  This completed Phase 1/2 trial from UCSF evaluated a conditioning regimen of busulfan, antithymocyte globulin, and fludarabine followed by d…

• CLINICAL_TRIALS trial_result · 2012-11-12
  Clinical Trial Update: Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease [Phase 1]

  Phase 1 trial evaluating donor stem cell transplantation in young patients with congenital amegakaryocytic thrombocytopenia, leukemia, myelo…