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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Cancer in Inherited Bone Marrow Failure SyndromesBone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Diamond-Blackfan anemia, Digestive System Diseases, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, Skin Abnormalities, Skin and Connective Tissue Diseases, X-linked disease, anemia, aplastic anemia, bone marrow disorder, dyskeratosis congenita, exocrine pancreatic insufficiency, hematologic disorder, hereditary disease, hereditary skin disorder, inherited aplastic anemia, lipomatosis, metabolic disease, pancreas disorder, pure red-cell aplasia, skin disorderRecruitingNot stated2026-10-08NCT00027274
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsAbnormalities, Multiple, Barth syndrome, Bone Marrow Failure Disorders, Cardiovascular Abnormalities, Cohen syndrome, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Digestive System Diseases, Hemic and Lymphatic Diseases, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, X-linked disease, agranulocytosis, autoimmune disease, bone marrow disorder, cardiovascular disorder, congenital heart disease, exocrine pancreatic insufficiency, heart disorder, hematologic disorder, hereditary disease, immune system disorder, inborn errors of metabolism, leukocyte disorder, leukopenia, lipomatosis, metabolic disease, neutropenia, pancreas disorderRecruitingPhase 32026-08-06NCT06056297
Shwachman Diamond Syndrome Registry and StudyBone 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, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neoplasms by Histologic Type, Nutritional and Metabolic Diseases, Shwachman-Diamond syndrome, agranulocytosis, bone marrow disorder, exocrine pancreatic insufficiency, hematologic disorder, leukemia, leukocyte disorder, leukopenia, lipomatosis, metabolic disease, neoplasm, neutropenia, pancreas disorderRecruitingNot stated2026-04-06NCT06056908
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationAIDS, Blood-Borne Infections, Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Diamond-Blackfan anemia, Digestive System Diseases, Familial Hemophagocytic Lymphocytosis, Genital Diseases, HIV infectious disease, Hemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, Infections, Kostmann syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lymphohistiocytosis, Hemophagocytic, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, RNA Virus Infections, Shwachman-Diamond syndrome, Slow Virus Diseases, Urogenital Diseases, anemia, aplastic anemia, bone marrow disorder, exocrine pancreatic insufficiency, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, histiocytosis, immune system disorder, immunoproliferative disorder, inborn error of immunity, infectious disease, inherited aplastic anemia, inherited hemoglobinopathy, interstitial lung disease, lentivirus infection, lipomatosis, lung disorder, lymphatic system disorder, lymphoproliferative syndrome, macrophage activation syndrome, metabolic disease, non-Langerhans cell histiocytosis, pancreas disorder, pure red-cell aplasia, respiratory system disorder, sexually transmitted disease, sickle cell disease, sickle cell-beta-thalassemia disease syndrome, uterine carcinosarcoma, viral infectious disease, viral sexually transmitted diseaseActive, not recruitingPhase 1 / Phase 22025-08-14NCT01966367
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