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ClinicalTrials.gov public records Last synced Sep 30, 2026, 9:11 AM EDT

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Showing 25–48 of 158 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Bone Marrow Failure Syndrome Clear all
Terminated Phase 2 Interventional Results available

Antithymocyte Globulin, Clofarabine, and Rituximab in Treating Patients After an Unsuccessful Stem Cell Transplant

NCT00617929
View directory record Official record
Conditions
Cancer
Interventions
anti-thymocyte globulin, rituximab, clofarabine, stem cell transplantation
Biological · Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Sep 30, 2026, 9:11 AM EDT
Completed Phase 2 Interventional

Hematopoietic Stem Cell Transplant for Fanconi Anemia

NCT01071239
View directory record Official record
Conditions
Fanconi Anemia
Interventions
CliniMACs device, Busulfan, Fludarabine, Cyclophosphamide, ATG
Device · Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 11, 2019 · Synced Sep 30, 2026, 9:11 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita

NCT01001598
View directory record Official record
Conditions
Fanconi Anemia, Dyskeratosis Congenita
Interventions
danazol
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 19, 2019 · Synced Sep 30, 2026, 9:11 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 Sep 30, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Active, not recruiting Phase 2 Interventional

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

NCT01659606
View directory record Official record
Conditions
Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
Interventions
alemtuzumab, Fludarabine, Cyclosporins, Mycophenolate mofetil, Tacrolimus
Biological · Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
30 Days to 65 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2034
U.S. locations
11
States / cities
Chicago, Illinois • Boston, Massachusetts • Kansas City, Missouri + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Recruiting Phase 1Phase 2 Interventional

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT03128996
View directory record Official record
Conditions
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders, Hemoglobinopathies, Non-malignant Disorders
Interventions
RIC regimen, GVHD prophylaxis regimen
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
1 Day to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2033
U.S. locations
4
States / cities
New Haven, Connecticut • Wilmington, Delaware • Grand Rapids, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 29, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Sep 30, 2026, 9:11 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 10, 2020 · Synced Sep 30, 2026, 9:11 AM EDT
Recruiting Phase 1 Interventional

Nucleoside Therapy in Patients With Telomere Biology Disorders

NCT06817590
View directory record Official record
Conditions
Telomere Biology Disorders, Dyskeratosis Congenita, Revesz Syndrome, Hoyeraal Hreidarsson Syndrome, Telomere Biology Disorders With Bone Marrow Failure, Interstitial Lung Disease Due to Systemic Disease (Telomere Biology Disorder), Pulmonary Fibrosis, Familial (Telomere Biology Disorder)
Interventions
deoxycytidine, deoxythymidine
Drug
Lead sponsor
Suneet Agarwal
Other
Eligibility
1 Year to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

Post T-plant Infusion of Allogeneic Cytokine Induced Killer (CIK) Cells as Consolidative Therapy in Myelodysplastic Syndromes/Myeloproliferative Disorders

NCT01392989
View directory record Official record
Conditions
Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure, Myelodysplastic Syndromes (MDS), Myeloproliferative Disorders
Interventions
CIK cells, Cyclosporine, Mycophenolate Mofetil, Thymoglobulin, Total Lymphoid Irradiation (TLI)
Drug · Radiation
Lead sponsor
Everett Meyer
Other
Eligibility
50 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated May 7, 2019 · Synced Sep 30, 2026, 9:11 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155
View directory record Official record
Conditions
Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
375 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2031
U.S. locations
14
States / cities
Duarte, California • Gainesville, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

NCT00455312
View directory record Official record
Conditions
Dyskeratosis Congenita, Aplastic Anemia
Interventions
Campath 1H, Cyclophosphamide, Fludarabine, Total Body Irradiation, Stem Cell Transplantation, antithymocyte globulin, Methylprednisolone
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 70 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 5, 2017 · Synced Sep 30, 2026, 9:11 AM EDT
Active, not recruiting Phase 2 Interventional

Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant

NCT03924401
View directory record Official record
Conditions
Graft Versus Host Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 20 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Wilmington, Delaware • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2026 · Synced Sep 30, 2026, 9:11 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 24, 2005 · Synced Sep 30, 2026, 9:11 AM EDT
Terminated Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

NCT00358657
View directory record Official record
Conditions
Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Sirolimus, Tacrolimus, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
3
States / cities
Nashville, Tennessee • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Sep 30, 2026, 9:11 AM 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 Sep 30, 2026, 9:11 AM EDT
Active, not recruiting Not applicable Interventional

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

NCT03047746
View directory record Official record
Conditions
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
Interventions
CliniMACs
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 25 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Sep 30, 2026, 9:11 AM EDT
Completed Phase 2 Interventional Results available

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

NCT03476330
View directory record Official record
Conditions
Fanconi Anemia, Squamous Cell Carcinoma
Interventions
Quercetin (dietary supplement)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Sep 30, 2026, 9:11 AM 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 Sep 30, 2026, 9:11 AM EDT
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 20, 2017 · Synced Sep 30, 2026, 9:11 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

NCT00005892
View directory record Official record
Conditions
Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
Interventions
cyclophosphamide, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 54 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Sep 30, 2026, 9:11 AM EDT
Recruiting Phase 2 Interventional

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

NCT04099966
View directory record Official record
Conditions
Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
Interventions
alpha beta depletion
Drug
Lead sponsor
Mitchell Cairo
Other
Eligibility
1 Day to 30 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 8, 2025 · Synced Sep 30, 2026, 9:11 AM EDT
Not listed Phase 1Phase 2 Interventional

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

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Sep 30, 2026, 9:11 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

NCT01464164
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
Sotatercept, Sotatercept with prednisone boost
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2021
U.S. locations
1
States / cities
Manhasset, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Sep 30, 2026, 9:11 AM EDT