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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:35 AM EDT

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Showing 1–24 of 352 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Bone Marrow Failure Disorders Clear all
Completed Phase 1Phase 2 Interventional Results available

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

NCT00630253
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine, Hematopoietic Stem Cell Transplantation, Methylprednisolone, Filgrastim, Cyclosporine, Mycophenolate Mofetil
Biological · Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 59 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 1Phase 2 Interventional

Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Donor

NCT00144729
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Bone marrow transplant
Procedure
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2007
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 30, 2008 · Synced Oct 21, 2026, 1:35 AM EDT
Active, not recruiting Phase 1 Interventional

A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

NCT03773393
View directory record Official record
Conditions
Bone Marrow Disease
Interventions
CK0801
Biological
Lead sponsor
Cellenkos, Inc.
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
Santa Monica, California • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 13, 2025 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 1 Interventional

Gene Therapy for the Treatment of Fanconi's Anemia Type C

NCT00001399
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 1:35 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 Oct 21, 2026, 1:35 AM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 13, 2014 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer

NCT00003816
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Nonmalignant Neoplasm, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
anti-thymocyte globulin, busulfan, carboplatin, cyclophosphamide, etoposide, fludarabine phosphate, melphalan, thiotepa, total-body irradiation
Biological · Drug · Radiation
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
4 Years to 70 Years
Enrollment
361 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Aug 13, 2021 · Synced Oct 21, 2026, 1:35 AM EDT
Terminated Not applicable Interventional

Blood Transplantation for Patients With Hematologic Malignancies or Bone Marrow Failure States

NCT00038779
View directory record Official record
Conditions
Leukemia, Non Hodgkin's Lymphoma, Aplastic Anemia
Interventions
Megadose T-cell Depleted HLA-nonidentical Blood Progenitor Cell Transplantation
Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2004
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 30, 2018 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Vosaroxin for Intermediate 2 or High-risk MDS After Failure With Hypomethylating Agent-based Therapy

NCT01980056
View directory record Official record
Conditions
Myelodysplastic Syndrome
Interventions
Vosaroxin
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 19, 2019 · Synced Oct 21, 2026, 1:35 AM EDT
Available No phase listed Expanded access

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

NCT03145545
View directory record Official record
Conditions
Leukemia, Bone Marrow Failure Syndrome, Immunodeficiencies
Interventions
Apha/beta T and CD19+ cell depletion using CliniMACS device
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month and older
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 21, 2026, 1:35 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 11, 2014 · Synced Oct 21, 2026, 1:35 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 7, 2011 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional

Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

NCT00004323
View directory record Official record
Conditions
Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, mesna, methotrexate, methylprednisolone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:35 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
10
States / cities
Duarte, California • Palo Alto, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Not applicable Interventional

Stem Cell Transplantation (SCT) for Genetic Diseases

NCT00004378
View directory record Official record
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting Phase 2 Interventional

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

NCT01174108
View directory record Official record
Conditions
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Interventions
Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
Device · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
2
States / cities
Baltimore, Maryland • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia

NCT00260689
View directory record Official record
Conditions
Immunosuppresion, Thrombocytopenia, Pancytopenia, Neutropenia
Interventions
Anti-thymocyte globulin (rabbit), Anti-thymocyte globulin (horse), Cyclosporine, Alemtuzumab
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 8, 2017 · Synced Oct 21, 2026, 1:35 AM EDT
Suspended Phase 1 Interventional

Donor Bone Marrow Derived Mesenchymal Stem Cells in Controlling Heart Failure in Patients With Cardiomyopathy Caused by Anthracyclines

NCT02962661
View directory record Official record
Conditions
Cardiomyopathy, Heart Failure, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
Interventions
Best Practice, Laboratory Biomarker Analysis, Mesenchymal Stem Cell Transplantation
Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 80 Years
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 21, 2026, 1:35 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 Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional Results available

Chemotherapy, Radiation Therapy, and Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer

NCT00003270
View directory record Official record
Conditions
Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions
anti-thymocyte globulin, filgrastim, busulfan, cyclophosphamide, cyclosporine, methylprednisolone, bone marrow ablation with stem cell support, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
5 Years to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2017
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Dec 13, 2019 · Synced Oct 21, 2026, 1:35 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 27, 2026 · Synced Oct 21, 2026, 1:35 AM EDT
Completed Phase 2 Interventional

Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

NCT00011505
View directory record Official record
Conditions
Diamond Blackfan Anemia
Interventions
G-CSF, Leukapheresis
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 21, 2026, 1:35 AM EDT
Withdrawn Not applicable Interventional

Unrelated HSCT in Patients With Fanconi Anemia

NCT02127905
View directory record Official record
Conditions
Fanconi Anemia
Interventions
CD34+ selected cells
Biological
Lead sponsor
Neena Kapoor, M.D.
Other
Eligibility
8 Weeks to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 11, 2017 · Synced Oct 21, 2026, 1:35 AM EDT
Recruiting Phase 2 Interventional

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

NCT01624805
View directory record Official record
Conditions
Aplastic Anemia, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome
Interventions
Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, Methylprednisolone, Pegfilgrastim
Biological · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2029
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 21, 2026, 1:35 AM EDT