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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Clinical Genetics Branch Eligibility Screening SurveyAbnormalities, Multiple, Bone Marrow Failure Disorders, Congenital Abnormalities, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Costello syndrome, Craniofacial Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Legius syndrome, Li-Fraumeni syndrome, Musculoskeletal Abnormalities, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, Thoracic Neoplasms, anemia, aplastic anemia, bone marrow disorder, cardiofaciocutaneous syndrome, chordoma, hematologic disorder, hereditary disease, hereditary neoplastic syndrome, inherited aplastic anemia, lung neoplasm, melanocytic neoplasm, melanoma, metabolic disease, mixed neoplasm, musculoskeletal system disorder, neoplasm, neuroendocrine neoplasm, primitive neuroectodermal tumor, pulmonary blastoma, respiratory tract neoplasm, skin disorder, skin neoplasm, susceptibility to familial cutaneous melanomaNot yet recruitingNot stated2026-10-09NCT07005297
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative NeoplasmsBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Myeloproliferative Disorders, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasiaRecruitingPhase 1 / Phase 22026-10-09NCT06303193
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 DepletionBone Marrow Failure Disorders, Chronic Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Lymphohistiocytosis, Hemophagocytic, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Primary Immunodeficiency Diseases, acute myeloid leukemia, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, histiocytosis, immune system disorder, immunoproliferative disorder, inborn error of immunity, inherited hemoglobinopathy, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Langerhans cell histiocytosisActive, not recruitingNot applicable2026-10-08NCT05236764
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
Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive TherapyBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderActive, not recruitingPhase 22026-10-06NCT02979873
Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Bone Marrow or Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific AntibodiesBlood Protein Disorders, Bone Marrow Failure Disorders, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Hodgkins lymphoma, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, acute myeloid leukemia, anemia, aplastic anemia, bone marrow disorder, cardiovascular disorder, hematologic disorder, hematopoietic and lymphoid system neoplasm, hemorrhagic disease, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, monoclonal gammopathy, myelodysplastic syndrome, myeloid leukemia, neoplasm, non-Hodgkin lymphoma, plasma cell neoplasm, primary myelofibrosis, vascular disorder, vascular hemostatic diseaseRecruitingEarly phase 12026-10-05NCT06398457
Experience and Management of Cancer Screening-Related Anxiety in Fanconi AnemiaBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease, neoplasmActive, not recruitingNot stated2026-10-02NCT06744283
Defining the Natural History of Squamous Cell Carcinoma in Fanconi AnemiaBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic diseaseRecruitingNot stated2026-10-01NCT05687149
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant OutcomeBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, myelodysplastic syndromeRecruitingPhase 1 / Phase 22026-10-01NCT03173937
Natural History of Acquired and Inherited Bone Marrow Failure SyndromesBone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Infant, Newborn, Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderRecruitingNot stated2026-09-30NCT05012111
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)Bone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderActive, not recruitingPhase 32026-09-25NCT05600426
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cellsBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndrome, myelodysplastic syndrome with excess blasts, myelodysplastic syndrome with single lineage dysplasia, pure red-cell aplasiaRecruitingPhase 22026-09-22NCT01174108
Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic AnemiaBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderNot yet recruitingNot stated2026-09-21NCT07819812
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic AnemiaBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderRecruitingPhase 1 / Phase 22026-09-21NCT07714512
Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRSBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, autoimmune disease, autoimmune hemolytic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, immune system disorder, myelodysplastic syndromeRecruitingNot stated2026-09-18NCT07578675
Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia PatientsBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderNot yet recruitingPhase 32026-09-16NCT07737262
Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic AnemiaBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderRecruitingNot applicable2026-09-16NCT07629947
Haploidentical HCT for Severe Aplastic AnemiaBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderRecruitingPhase 22026-09-16NCT04558736
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
Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral CancerBone Marrow Failure Disorders, Chemically-Induced Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Erythroplasia, Fanconi anemia, Head and Neck Neoplasms, Hemic and Lymphatic Diseases, Lichenoid Eruptions, Mouth Neoplasms, Neoplasms by Site, Nutritional and Metabolic Diseases, Skin Diseases, Papulosquamous, Skin and Connective Tissue Diseases, Stomatognathic Diseases, anemia, aplastic anemia, bone marrow disorder, erythroplakia, hematologic disorder, hereditary disease, hereditary mucosal leukokeratosis, hereditary skin disorder, inherited aplastic anemia, lichen planus, metabolic disease, mouth disorder, neoplasm, nicotine dependence, precancerous condition, psychiatric disorder, skin disorder, substance-related disorderActive, not recruitingNot applicable2026-09-14NCT00542373
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNIBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, acute leukemia, anemia, aplastic anemia, bone marrow disorder, graft versus host disease, hematologic disorder, immune system disorder, myelodysplastic syndromeNot yet recruitingPhase 1 / Phase 22026-09-09NCT07796620
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD ProphylaxisBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, Hemoglobinuria, Paroxysmal, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hemolytic anemia, myelodysplastic syndromeRecruitingPhase 22026-09-08NCT03520647
The HOPE Biobank Resource (BMT CTN 2402 HOPE)Bone Marrow Failure, Bone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited hemoglobinopathyRecruitingNot stated2026-08-27NCT07227155
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard TreatmentBone Marrow Failure Disorders, Disease Attributes, Hemic and Lymphatic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, anemia, aplastic anemia, bone marrow disorder, disease, hematologic disorderNot yet recruitingPhase 2 / Phase 32026-08-26NCT07786922
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow FailureBone Marrow Failure Disorders, Hemic and Lymphatic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorderActive, not recruitingPhase 1 / Phase 22026-08-18NCT05998408