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Showing 1–24 of 267 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Bone Marrow Aplasia Clear all
Recruiting No phase listed Observational Accepts healthy volunteers

Cancer in Inherited Bone Marrow Failure Syndromes

NCT00027274
View directory record Official record
Conditions
Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome, Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
2
States / cities
Bethesda, Maryland • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

NCT00987480
View directory record Official record
Conditions
Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
Interventions
Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
Drug · Device
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
6
States / cities
Boston, Massachusetts • New York, New York • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Jul 22, 2026, 8:47 AM EDT
Recruiting Phase 2 Interventional Results available

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

NCT04304820
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 99 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes

NCT07712562
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
Interventions
Dibotatug (DR-01)
Drug
Lead sponsor
Dren Bio
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
9
States / cities
Duarte, California • Palo Alto, California • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Completed No phase listed Observational

Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia

NCT01146210
View directory record Official record
Conditions
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a), Childhood Acute Monocytic Leukemia (M5b), Childhood Acute Myeloblastic Leukemia With Maturation (M2), Childhood Acute Myeloblastic Leukemia Without Maturation (M1), Childhood Acute Myelomonocytic Leukemia (M4), Childhood Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Fanconi Anemia, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Ringed Sideroblasts, Secondary Myelodysplastic Syndromes, Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions
laboratory biomarker analysis
Other
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Monrovia, California
Source: ClinicalTrials.gov public record
Updated May 16, 2016 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Not applicable Interventional

Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia

NCT00186797
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Allogeneic stem cell transplant, Fludarabine, Cyclophosphamide
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 8, 2010 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional

Medical Treatment for Diamond Blackfan Anemia

NCT00001749
View directory record Official record
Conditions
Fanconi's Anemia, Hematologic Disease
Interventions
Antithymocyte globulin, Cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 8:47 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

NCT05998408
View directory record Official record
Conditions
Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL, Hypoplastic MDS
Interventions
Ruxolitinib
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia

NCT01187017
View directory record Official record
Conditions
Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
Interventions
Cyclophosphamide, Fludarabine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2021 · Synced Jul 22, 2026, 8:47 AM EDT
Recruiting Phase 2 Interventional

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

NCT04232085
View directory record Official record
Conditions
Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita, Telomere Biology Disorder, Short Telomere Syndrome
Interventions
Alemtuzumab, Fludarabine, Melphalan, Low Dose Total Body Irradiation, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
4 Months to 50 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 27, 2025 · Synced Jul 22, 2026, 8:47 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones

NCT00556400
View directory record Official record
Conditions
Aplastic Anemia, Menorrhagia, Amenorrhea
Interventions
Lo-Ovral Oral Contraceptive Pills, Placebo - sugar pill
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
12 Years to 55 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 18, 2013 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 3 Interventional Results available

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

NCT00235391
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 6, 2011 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional Results available

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

NCT00053989
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Fanconi Anemia, Aplastic Anemia
Interventions
anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim, therapeutic allogeneic lymphocytes, cyclophosphamide, fludarabine phosphate, methylprednisolone, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 75 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2018
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Feb 9, 2020 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders

NCT00533923
View directory record Official record
Conditions
AML, ALL, CLL, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma, Multiple Myeloma, Aplastic Anemia, Myeloproliferative Disorder
Interventions
Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF
Drug
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
Up to 65 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2007
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 17, 2016 · Synced Jul 22, 2026, 8:47 AM EDT
Recruiting No phase listed Observational

Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)

NCT07186179
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Mobilization Regimen
Drug
Lead sponsor
Northwell Health
Other
Eligibility
3 Years to 30 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New Hyde Park, New York
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional Results available

A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients

NCT00922883
View directory record Official record
Conditions
Anemia, Aplastic, Anemia, Hypoplastic, Thrombocytopenia
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
12 Years and older
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Phase 2 Interventional Results available

Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies

NCT01760655
View directory record Official record
Conditions
Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM, Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214, Aplastic Anemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Follicular Lymphoma, Hodgkin Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Myeloid Leukemia, Non-Hodgkin Lymphoma, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Therapy-Related Acute Myeloid Leukemia
Interventions
Fludarabine phosphate, Busulfan, Total-Body Irradiation, Therapeutic Allogeneic Lymphocytes, Cyclophosphamide, Allogeneic Hematopoietic Stem Cell Transplantation, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Mycophenolate Mofetil, Laboratory Biomarker Analysis
Drug · Radiation · Biological + 2 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
Not listed
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Jul 22, 2026, 8:47 AM EDT
Completed Not applicable Interventional Results available

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

NCT00244010
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced Jul 22, 2026, 8:47 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia

NCT00590460
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia
Interventions
CAMPATH-1H, Anti-CD45, Fludarabine, Stem cell infusion
Biological · Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 23, 2018 · Synced Jul 22, 2026, 8:47 AM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Jul 22, 2026, 8:47 AM EDT
Recruiting No phase listed Observational

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

NCT07649031
View directory record Official record
Conditions
Fanconi Anemia
Interventions
MRIs
Diagnostic Test
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

NCT00001962
View directory record Official record
Conditions
Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
Interventions
Daclizumab
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 8, 2021 · Synced Jul 22, 2026, 8:47 AM EDT
Active, not recruiting Not applicable Interventional

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

NCT00542373
View directory record Official record
Conditions
Erythroplakia, Fanconi Anemia, Lichen Planus, Oral Cavity and Lip Precancerous Condition, Oral Cavity Leukoplakia, Oral Neoplasm, Premalignant Lesion, Tobacco Use Disorder
Interventions
Cytology Specimen Collection Procedure, Diagnostic Microscopy, Diagnostic Procedure, Fluorescence Imaging, Fluorescence Spectroscopy
Other · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Jul 22, 2026, 8:47 AM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2021 · Synced Jul 22, 2026, 8:47 AM EDT