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ClinicalTrials.gov public records Last synced Oct 27, 2026, 10:22 PM EDT

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Showing 1–24 of 319 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Bone Marrow Failure Syndromes Clear all
Terminated Not applicable Interventional Results available

G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome

NCT00228813
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
Granulocyte Colony Stimulating Factor
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 22 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2015
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 11, 2017 · Synced Oct 27, 2026, 10:22 PM 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 2, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Completed Phase 2 Interventional Results available

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

NCT01231841
View directory record Official record
Conditions
Aplastic Anemia
Interventions
cyclosporine, anti-thymocyte globulin
Drug · Biological
Lead sponsor
The Cleveland Clinic
Other
Eligibility
12 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 22, 2023 · Synced Oct 27, 2026, 10:22 PM EDT
Terminated Early Phase 1 Interventional

New York Blood Center National Cord Blood Program

NCT00212407
View directory record Official record
Conditions
Leukemia, Lymphoma, Genetic Disease, Severe Aplastic Anemia, Myelodysplasia
Interventions
Umbilical Cord Blood Transplantation
Biological
Lead sponsor
New York Blood Center
Other
Eligibility
Not listed
Enrollment
4,476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 2, 2018 · Synced Oct 27, 2026, 10:22 PM EDT
Terminated Phase 3 Interventional

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

NCT05600426
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG), rabbit anti-thymocyte globulin (ATG), Methotrexate, Fludarabine, Cyclophosphamide, low-dose total body irradiation (TBI), Immunosuppressive Therapy (IST)
Drug · Procedure · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Years to 25 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 44 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Completed No phase listed Observational

Tissue Sample Collection From Patients With Fanconi Anemia

NCT00899522
View directory record Official record
Conditions
Fanconi Anemia, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
biologic sample preservation procedure, biopsy
Other · Procedure
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
1 Year to 55 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2013
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 4, 2017 · Synced Oct 27, 2026, 10:22 PM EDT
Completed Phase 2 Interventional Results available

Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases

NCT02277639
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Immunodeficiencies, Immune Dysregulation Syndromes
Interventions
CliniMACs device
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 22 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 11, 2018 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Role of Acetaldehyde in the Development of Oral Cancer

NCT05973656
View directory record Official record
Conditions
Alcohol-Related Carcinoma, Fanconi Anemia, Oral Cavity Carcinoma
Interventions
Alcohol, Biospecimen Collection, Breath Test
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
18 Years to 45 Years
Enrollment
170 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

NCT00578903
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Cytoxan, Campath, Total Body Irradiation (TBI), FK-506, Methotrexate, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Minute to 60 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 22, 2016 · Synced Oct 27, 2026, 10:22 PM 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 9, 2021 · Synced Oct 27, 2026, 10:22 PM EDT
Completed No phase listed Observational

Investigation of the Cylex® ImmuKnow® Assay

NCT00569842
View directory record Official record
Conditions
Leukemia, Non-Hodgkin's Lymphoma, Chronic Lymphocytic Leukemia, Hodgkin's Disease, Multiple Myeloma, Myelodysplastic Syndromes, Myeloproliferative Disorders, Aplastic Anemia, Chronic Myelogenous Leukemia
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Sep 11, 2014 · Synced Oct 27, 2026, 10:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

NCT00595127
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
Drug · Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2016 · Synced Oct 27, 2026, 10:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

NCT05828108
View directory record Official record
Conditions
Steroid-refractory Diamond-Blackfan Anemia (DBA)
Interventions
Bitopertin
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting No phase listed Observational

The Role of Brain-Bone Marrow-Gut Interaction Following Major Trauma

NCT06606119
View directory record Official record
Conditions
Trauma Injury, Trauma, Critical Illness, Microbiome, Chronic Anemia, Acute Blood Loss Anemia
Interventions
Data and tissue collection
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 100 Years
Enrollment
275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 6, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

NCT03333486
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
Drug · Other · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
1 Year to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Jul 4, 2025 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting Phase 1 Interventional

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

NCT07476183
View directory record Official record
Conditions
RPS19 Deficient Diamond-Blackfan Anemia
Interventions
APR-2020
Biological
Lead sponsor
Apriligen, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Stanford, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Not listed Not applicable Interventional

Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia

NCT00017654
View directory record Official record
Conditions
Graft Versus Host Disease, Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, methylprednisolone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Northwestern Memorial Hospital
Other
Eligibility
15 Years to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 27, 2026, 10:22 PM EDT
Terminated Phase 2 Interventional Results available

Low-Dose Decitabine in Myelodysplastic Syndrome Post Azacytidine Failure

NCT00113321
View directory record Official record
Conditions
Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia
Interventions
Decitabine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 7, 2012 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting Phase 2 Interventional

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors

NCT05194397
View directory record Official record
Conditions
Lymphoblastic Leukemia, Cancer Survivors, Aplastic Anemia, Myelodysplastic Syndromes, Myeloid Leukemia
Interventions
Nicotinamide Riboside, Placebo, Exercise Intervention
Dietary Supplement · Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
10 Years to 30 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Duarte, California • Philadelphia, Pennsylvania • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting No phase listed Observational

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

NCT01351545
View directory record Official record
Conditions
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
Interventions
A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Drug
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
Not listed
Enrollment
99,999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2041
U.S. locations
142
States / cities
Birmingham, Alabama • Gilbert, Arizona • Phoenix, Arizona + 90 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting Phase 2 Interventional

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

NCT06517641
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Haploidentical donor bone marrow transplant, Unrelated donor bone marrow transplant
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
3 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
25
States / cities
Birmingham, Alabama • Duarte, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 27, 2026, 10:22 PM EDT
Completed Phase 2 Interventional Results available

Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure

NCT02159872
View directory record Official record
Conditions
Leukemia
Interventions
Omacetaxine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Oct 27, 2026, 10:22 PM EDT
Recruiting Not applicable Interventional

Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

NCT02958462
View directory record Official record
Conditions
Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia, Bone Marrow Failure Syndrome, Clonal Cytopenia of Undetermined Significance, Clonal Hematopoiesis of Indeterminate Potential, Hematologic Neoplasms, Hematopoietic and Lymphatic System Neoplasm, Hereditary Neoplastic Syndrome, Idiopathic Cytopenia of Undetermined Significance, Idiopathic Dysplasia of Uncertain Significance, Low Risk Myelodysplastic Syndrome
Interventions
Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy, Buccal Swab, Clinical Evaluation, Genetic Counseling, Quality-of-Life Assessment, Electronic Health Record Review
Procedure · Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2035
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 23, 2026 · Synced Oct 27, 2026, 10:22 PM 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 27, 2026 · Synced Oct 27, 2026, 10:22 PM EDT