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Bone Marrow Failure Disorders × Clear all

NCT06839456 2026-04-15

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

Classical hematologyLymphoidOther hematologic neoplasm

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Anemia, Refractory, with Excess of Blasts, Hemoglobinopathies, Immunologic Deficiency Syndromes, Leukemia, Lymphoma, Non-Hodgkin, Metabolism, Inborn Errors

NCT06056908 2026-04-06

SDSR

Recruiting

5,000 enrolled

Classical hematologyOther hematologic neoplasm

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Shwachman-Diamond Syndrome, Leukemia, Neutropenia

NCT04644016 2026-03-13

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

Phase 2 Recruiting

31 enrolled

Classical hematologyLymphoidMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Anemia, Refractory, with Excess of Blasts, Hemoglobinopathies, Lymphoma, Non-Hodgkin, Myeloproliferative Disorders, Leukemia, Myeloid, Acute

NCT04965597 2026-03-09

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Phase 2 Completed

40 enrolled

Classical hematology

Congenital amegakaryocytic thrombocytopenia, Bone Marrow Failure Disorders, Shwachman-Diamond Syndrome, Anemia, Hypochromic, Hemoglobinuria, Paroxysmal, Anemia, Diamond-Blackfan

NCT02958462 2026-02-23

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Phase NA Recruiting

2,000 enrolled

Classical hematologyOther hematologic neoplasm

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Cytopenia, Neoplastic Syndromes, Hereditary, Hematologic Neoplasms

NCT02356653 2026-02-20

ExpMACs

Phase EARLY_PHASE1 Recruiting

100 enrolled

Other hematologic neoplasm

Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome, Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes, Leukemia, Metabolism, Inborn Errors

NCT07408583 2026-02-19

Prenatal Transplantation for Fetuses With Fanconi Anemia

Phase 1/2 Not yet recruiting

12 enrolled

Classical hematology

Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Fanconi Anemia, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Anemia, Hypoplastic, Congenital, Genetic Diseases, Inborn, DNA Repair-Deficiency Disorders

NCT03622788 2025-12-17

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

Phase 1/2 Active not recruiting

16 enrolled

Classical hematologyLymphoidMyeloidOther hematologic neoplasmPlasma cell

Bone Marrow Failure Disorders, Anemia, Aplastic, Hodgkin Disease, Lymphoma, Follicular, Lymphoma, Non-Hodgkin, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma

NCT06430788 2025-12-17

A Study of Emapalumab for Pediatric Aplastic Anemia

Phase 2 Recruiting

35 enrolled

Classical hematology

Bone Marrow Failure Disorders, Cytopenia, Anemia, Aplastic

NCT05236764 2025-10-30

HAPLOTAB

Phase NA Active not recruiting

3 enrolled 9 charts

Classical hematologyLymphoidMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Primary Immunodeficiency Diseases, Cytopenia, Anemia, Aplastic, Hemoglobinopathies, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Lymphohistiocytosis, Hemophagocytic, Precursor Cell Lymphoblastic Leukemia-Lymphoma

NCT06940570 2025-10-23

MATCH

Phase 3 Suspended

60 enrolled

Classical hematologyOtherOther hematologic neoplasm

Bone Marrow Failure Disorders, Anemia, Aplastic, Leukemia, Neoplasms, Pain, Intractable, Mucositis

NCT04618081 2025-07-08

Revlimid® Capsules General Drug Use-results Surveillance (Relapsed or Refractory FL and MZL)

Completed

151 enrolled

Lymphoid

Bone Marrow Failure Disorders, Lymphoma, Lymphoma, Non-Hodgkin, Neutropenia

NCT02722668 2025-07-04

UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep

Phase 2 Completed

15 enrolled 15 charts

LymphoidMyeloidOther hematologic neoplasmPlasma cellSarcoma

Bone Marrow Failure Disorders, Burkitt Lymphoma, Hodgkin Disease, Leukemia, Leukemia, Plasma Cell, Lymphoma, Follicular, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Prolymphocytic, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Lymphoma, T-Cell, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Dendritic Cell Sarcoma, Interdigitating, Primary Myelofibrosis

NCT02988466 2025-05-28

Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haplo-HCT

Phase 2 Completed

78 enrolled 16 charts

LymphoidMyeloidOther hematologic neoplasmPlasma cellSarcoma

Bone Marrow Failure Disorders, Burkitt Lymphoma, Hodgkin Disease, Leukemia, Lymphoma, Follicular, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Lymphoma, T-Cell, Neoplasm, Residual, Lymphoma, B-Cell, Marginal Zone, Hematologic Neoplasms, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Dendritic Cell Sarcoma, Interdigitating, Primary Myelofibrosis

NCT03145545 2025-05-22

Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

Available

Other hematologic neoplasm

Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes, Leukemia

NCT02061800 2024-06-04

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Phase 1/2 Active not recruiting

14 enrolled

Classical hematologyLymphoidMyeloidOther hematologic neoplasm

Neutropenia, Severe Congenital, Autosomal Recessive 3, Bone Marrow Failure Disorders, Shwachman-Diamond Syndrome, Primary Immunodeficiency Diseases, Acquired Immunodeficiency Syndrome, Anemia, Aplastic, Anemia, Sickle Cell, Hemoglobinopathies, Histiocytosis, Langerhans-Cell, Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Histiocytosis, Anemia, Diamond-Blackfan, Lymphohistiocytosis, Hemophagocytic, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myelomonocytic, Juvenile, Macrophage Activation Syndrome

NCT06250595 2024-02-09

ENROL

Recruiting

37,090 enrolled

Classical hematologyOther hematologic neoplasmPlasma cell

Bone Marrow Failure Disorders, Anemia, Anemia, Sickle Cell, Leukemia, Thalassemia, Iron Metabolism Disorders, Hematologic Neoplasms, Hemostatic Disorders, Neoplasms, Plasma Cell

NCT00968864 2022-04-22

T-cell Depleted Alternative Donor Transplantation

Phase 2 Terminated

53 enrolled 14 charts

LymphoidMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Hemoglobinopathies, Immunologic Deficiency Syndromes, Lymphoma, Myelodysplastic Syndromes, Osteopetrosis, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma

NCT01071226 2021-10-25

Peripheral Blood Stem Cell Transplantation Using the CliniMACS Device

Phase NA Completed

60 enrolled

LymphoidOther hematologic neoplasm

Bone Marrow Failure Disorders, Leukemia, Lymphoma

NCT01200017 2021-05-27

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

No longer available

Classical hematologyLymphoidMyeloidOther hematologic neoplasmOther neoplasm

Bone Marrow Failure Disorders, Cytopenia, Bone Diseases, Bone Diseases, Developmental, Bone Marrow Diseases, Graft vs Host Disease, Hematologic Diseases, Hemoglobinopathies, Immunologic Deficiency Syndromes, Immune System Diseases, Immunoproliferative Disorders, Leukemia, Leukemia, Lymphoid, Leukemia, Myeloid, Leukocyte Disorders, Lymphatic Diseases, Lymphoma, Lymphoproliferative Disorders, Musculoskeletal Diseases, Myelodysplastic Syndromes, Myeloproliferative Disorders, Neoplasms, Neoplasms by Histologic Type, Osteochondrodysplasias, Osteopetrosis, Osteosclerosis, Pancytopenia, Precancerous Conditions, Preleukemia, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Genetic Diseases, Inborn, Precursor Cell Lymphoblastic Leukemia-Lymphoma

NCT02358499 2020-12-22

Genetic Variation and Variability in Posaconazole Pharmacokinetics in Children

Phase 1 Completed

31 enrolled

Other hematologic neoplasm

Bone Marrow Failure Disorders, Leukemia, Mycoses

NCT01596699 2020-02-26

Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation

Phase 2 Terminated

16 enrolled 11 charts

Bone Marrow Failure Disorders, Metabolic Diseases

NCT02145039 2019-12-26

Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases

Phase NA Terminated

2 enrolled 11 charts

LymphoidMyeloidOther hematologic neoplasmPlasma cellSarcoma

Bone Marrow Failure Disorders, Burkitt Lymphoma, Hodgkin Disease, Leukemia, Lymphoma, Follicular, Multiple Myeloma, Myelodysplastic Syndromes, Leukemia, Lymphocytic, Chronic, B-Cell, Leukemia, Prolymphocytic, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Myeloid, Acute, Lymphoma, B-Cell, Marginal Zone, Lymphoma, Mantle-Cell, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Dendritic Cell Sarcoma, Interdigitating

NCT01049854 2018-08-21

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

Phase 2 Completed

20 enrolled

Classical hematologyLymphoidMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Anemia, Sickle Cell, Immunologic Deficiency Syndromes, Leukemia, Lymphoma, Metabolism, Inborn Errors, Histiocytosis, beta-Thalassemia

NCT03086252 2018-07-26

Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

Phase NA Withdrawn

Classical hematologyMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Anemia, Anemia, Aplastic, Leukemia, Myelodysplastic Syndromes, Hematologic Neoplasms

NCT00897260 2018-01-26

Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic Disorders

Phase NA Completed

27 enrolled

Other hematologic neoplasm

Bone Marrow Failure Disorders, Hematologic Neoplasms

NCT02337595 2016-04-22

45RA_NEG_DLI

Phase 1/2 Completed

30 enrolled

LymphoidOther hematologic neoplasm

Immune Deficiency Disease, Bone Marrow Failure Disorders, Graft vs Host Disease, Opportunistic Infections, Leukemia, Myeloid, Acute, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma

NCT01757145 2015-06-19

Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation

Phase 2 Unknown

20 enrolled

Other hematologic neoplasm

Bone Marrow Failure Disorders, Hematologic Neoplasms

NCT01050439 2015-04-15

Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

Phase 2 Terminated

22 enrolled

LymphoidMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Immunologic Deficiency Syndromes, Leukemia, Lymphoma, Histiocytosis

NCT00315419 2010-04-21

Identifying Characteristics of Bone Marrow Failure Syndromes

Unknown

450 enrolled

Classical hematologyMyeloidOther hematologic neoplasm

Bone Marrow Failure Disorders, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Leukemia, Lymphoid, Myelodysplastic Syndromes, Purpura, Thrombocytopenic, Red-Cell Aplasia, Pure, Leukemia, Large Granular Lymphocytic