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Showing 1–24 of 319 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Bone Marrow Failure Syndromes Clear all
Completed Phase 1 Interventional

Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia

NCT00093743
View directory record Official record
Conditions
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Childhood Acute Myeloid Leukemia in Remission, Childhood Myelodysplastic Syndromes, Fanconi Anemia, Previously Treated Myelodysplastic Syndromes
Interventions
fludarabine phosphate, cyclosporine, total-body irradiation, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, mycophenolate mofetil
Drug · Radiation · Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
5
States / cities
Chicago, Illinois • Indianapolis, Indiana • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 19, 2017 · Synced Jul 21, 2026, 9:33 AM EDT
Completed No phase listed Observational

Collection of Biological Samples From Patients With Hepatitis-Associated Aplastic Anemia

NCT00050115
View directory record Official record
Conditions
Hepatitis-Associated Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 7, 2021 · Synced Jul 21, 2026, 9:33 AM EDT
Terminated Phase 2 Interventional Results available

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

NCT01596699
View directory record Official record
Conditions
Myeloid Malignancy, Bone Marrow Failure Syndrome, Transfusion-dependent Red Blood Cell (RBC) Defect, Congenital Immunodeficiency, Metabolic Disease, Severe Immune Dysregulation
Interventions
Alemtuzumab, Busulfan, Fludarabine, Clofarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
3 Months to 30 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 25, 2020 · Synced Jul 21, 2026, 9:33 AM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

NCT06090669
View directory record Official record
Conditions
Inherited Bone Marrow Failure Syndrome, Familial Platelet Disorder With Predisposition to Myeloid Malignancies
Interventions
imatinib, TruSight Oncology
Drug · Device
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 120 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 21, 2026, 9:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

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 Outcome

NCT03173937
View directory record Official record
Conditions
Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
Interventions
Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
Biological · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 75 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 21, 2026, 9:33 AM EDT
Completed No phase listed Observational

Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609827
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2021 · Synced Jul 21, 2026, 9:33 AM EDT
Completed Phase 2 Interventional Results available

Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment

NCT00944749
View directory record Official record
Conditions
Anemia, Aplastic, Anemia, Hypoplastic
Interventions
h-ATG (ATGAM ), Cyclosporine (Gengraf )
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 82 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 22, 2021 · Synced Jul 21, 2026, 9:33 AM EDT
Terminated Phase 2 Interventional

Thrombosomes® in Bleeding Thrombocytopenic Patients Study

NCT04631211
View directory record Official record
Conditions
Thrombocytopenia, Hematologic Malignancy, Bone Marrow Aplasia, Myeloproliferative Disorders, Myelodysplastic Syndromes, Platelet Refractoriness
Interventions
Thrombosomes, Liquid Stored Platelets (LSP)
Biological
Lead sponsor
Cellphire Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
4
States / cities
Duarte, California • Washington D.C., District of Columbia • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated May 24, 2023 · Synced Jul 21, 2026, 9:33 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 21, 2026, 9:33 AM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

NCT00301834
View directory record Official record
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation
Biological · Drug · Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 21 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 27, 2017 · Synced Jul 21, 2026, 9:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

NCT04784052
View directory record Official record
Conditions
Fanconi Anemia
Interventions
JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide, Fludarabine, Rituximab
Drug · Device · Biological
Lead sponsor
Porteus, Matthew, MD
Other
Eligibility
2 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Jul 21, 2026, 9:33 AM EDT
Completed Phase 4 Interventional

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

NCT03176849
View directory record Official record
Conditions
Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder, Pediatric Acute Myeloid Leukemia, Pediatric Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Sickle Cell Anemia in Children, Aplastic Anemia, Thalassemia in Children
Interventions
Vitamin D3, Standard Vitamin D3 Supplementation
Dietary Supplement
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
1 Year to 25 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 16, 2020 · Synced Jul 21, 2026, 9:33 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

NCT00005898
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, fludarabine, methylprednisolone
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
0 Years to 34 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 9:33 AM EDT
Recruiting No phase listed Observational

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05012111
View directory record Official record
Conditions
Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2041
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Jul 21, 2026, 9:33 AM EDT
Completed No phase listed Observational

A Safety and Efficacy of Pomalyst® Capsules Under the Actual Use in All Patients Who Are Treated With Pomalyst at a Dose of 1 mg, 2 mg, 3 mg, or 4 mg

NCT02921828
View directory record Official record
Conditions
Multiple Myeloma
Interventions
Not listed
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
1,149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
1
States / cities
No City Provided, New Jersey
Source: ClinicalTrials.gov public record
Updated Jul 4, 2022 · Synced Jul 21, 2026, 9:33 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Jul 21, 2026, 9:33 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 21, 2026, 9:33 AM EDT
Completed Phase 1 Interventional Results available

Thrombosomes® in Bleeding Thrombocytopenic Patients

NCT03394755
View directory record Official record
Conditions
Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
Interventions
Thrombosomes
Biological
Lead sponsor
Cellphire Therapeutics, Inc.
Industry
Eligibility
18 Years to 74 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
6
States / cities
Duarte, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2023 · Synced Jul 21, 2026, 9:33 AM 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 10, 2014 · Synced Jul 21, 2026, 9:33 AM EDT
Completed Phase 2 Interventional

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

NCT00578435
View directory record Official record
Conditions
Genetic Disorders, Sickle Cell Anemia
Interventions
Busulfan, Cyclophosphamide, BMD
Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 11, 2008 · Synced Jul 21, 2026, 9:33 AM 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 9, 2016 · Synced Jul 21, 2026, 9:33 AM 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 16, 2026 · Synced Jul 21, 2026, 9:33 AM EDT
Not listed Phase 1 Interventional

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

NCT00006127
View directory record Official record
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 9:33 AM EDT
Completed Not applicable Interventional Results available

Unrelated Umbilical Cord Blood (UBC)Transplantation

NCT01768845
View directory record Official record
Conditions
Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome, Multiple Myeloma, Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Chronic Lymphocytic Leukemia (CLL), Acute Lymphocytic Leukemia (ALL), Severe Aplastic Anemia
Interventions
umbilical cord blood (UCB)
Genetic
Lead sponsor
West Virginia University
Other
Eligibility
16 Years to 70 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2021
U.S. locations
1
States / cities
Morgantown, West Virginia
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Jul 21, 2026, 9:33 AM EDT