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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Eltrombopag for Fanconi AnemiaBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Inflammation, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, pancytopeniaActive, not recruitingPhase 22026-09-16NCT03206086
Clinical, Hematological and Pathological Profile of PancytopeniaCytopenia, Hemic and Lymphatic Diseases, hematologic disorder, pancytopeniaNot yet recruitingNot stated2026-08-25NCT07785726
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure SyndromesBone Marrow Failure Disorders, Cytopenia, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, leukocyte disorder, pancytopeniaNot yet recruitingPhase 1 / Phase 22026-07-24NCT07712562
Eltrombopag With Standard Immunosuppression for Severe Aplastic AnemiaBone Marrow Failure Disorders, Cytopenia, Hemic and Lymphatic Diseases, anemia, aplastic anemia, autoimmune disease, blood platelet disease, bone marrow disorder, hematologic disorder, immune system disorder, pancytopenia, thrombocytopeniaActive, not recruitingPhase 1 / Phase 22026-04-13NCT01623167
Shwachman-Diamond Syndrome Global Patient Survey and Partnering PlatformBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Digestive System Diseases, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Inherited Immunodeficiency Diseases, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, Shwachman-Diamond syndrome, bone marrow disorder, exocrine pancreatic insufficiency, follicular lymphoma, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, immune system disorder, immunoproliferative disorder, inborn error of immunity, lipomatosis, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, metabolic disease, myelodysplastic syndrome, neoplasm, pancreas disorder, pancytopeniaRecruitingNot stated2025-05-31NCT06999954
Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, bone marrow disorder, hematologic disorder, pancytopeniaRecruitingNot stated2024-11-07NCT05196789