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Clinical Trials for Bone Marrow Failure Disorders

• Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency
• JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
• A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies
• Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders
• MNCs for Bone Marrow Failure After Malignant Diseases Children Chemotherapy
• The Use of Eltrombopag Post HSCT in BMFS
• HCT Using Treosulfan for Bone Marrow Failure Diseases (BMT CTN 1904)
• Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
• Haploidentical HCT for Severe Aplastic Anemia
• TPO-Mimetic Use in Children for Hemotopoietic Failure
• Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide
• Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant
• Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
• Lung and Bone Marrow Transplantation for Lung and Bone Marrow Failure
• Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
• NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
• UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
• Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
• Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
• Nandrolone Decanoate in the Treatment of Telomeropathies
• Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
• Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)
• Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation
• Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria
• Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
• Eltrombopag for Moderate Aplastic Anemia
• CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
• Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
• Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic Disorders
• Comparison of Cy-Atg Vs Cy-Flu-Atg for the Conditioning Therapy in Allo-HCT
• G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome
• Stem Cell Transplant for Bone Marrow Failure Syndromes
• Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes