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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Aplastic Anemias Clear all
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 12, 2014 · Synced Sep 28, 2026, 3:27 AM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

NCT07527975
View directory record Official record
Conditions
Fanconi Anemia
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2038
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Recruiting Early Phase 1 Interventional

Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies

NCT06398457
View directory record Official record
Conditions
Hematologic Malignancy, Bone Marrow Transplant Rejection, Acute Myeloid Leukemia (AML), Myelodysplastic Syndromes (MDS), Acute Lymphoblastic Leukemia (ALL), Adult, Multiple Myeloma, Aplastic Anemia, Lymphoma, Non Hodgkin Lymphoma, Hodgkin Lymphoma, Chronic Myeloid Leukemia, Myelofibrosis
Interventions
Darzalex Faspro (Daratumumab and hyaluronidase-fihj), JH-DSA Semi-Quant Screen and Response Score
Drug · Device
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Not applicable Interventional

Blood Cell Collection for Future Use in Individuals With Fanconi Anemia

NCT00271089
View directory record Official record
Conditions
Fanconi Anemia
Interventions
PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
Procedure · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Year to 35 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 11, 2016 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional Results available

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

NCT00453388
View directory record Official record
Conditions
Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome, Fanconi Anemia, Previously Treated Myelodysplastic Syndrome
Interventions
Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
4
States / cities
Oakland, California • Nashville, Tennessee • Seattle, Washington + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2020 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

NCT01044186
View directory record Official record
Conditions
Transfusional Iron Overload
Interventions
ICL670
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
8
States / cities
Oakland, California • Orange, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2017 · Synced Sep 28, 2026, 3:27 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 16, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional Results available

Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer

NCT00975975
View directory record Official record
Conditions
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Multiple Myeloma, Myelofibrosis, Anemia, Aplastic, Hemoglobinuria, Paroxysmal
Interventions
Basiliximab
Drug
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 25, 2016 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional

Combination Therapy of Severe Aplastic Anemia

NCT00001964
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Cyclosporine A, ATG, MMF
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
1 Year to 99 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 15, 2021 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

NCT02462252
View directory record Official record
Conditions
Aplastic Anemia, Hypoplastic Myelodysplastic Syndrome
Interventions
BL-8040, horse anti-thymocyte globulin (hATG), Methylprednisolone, Cyclosporine
Drug
Lead sponsor
BioLineRx, Ltd.
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 28, 2020 · Synced Sep 28, 2026, 3:27 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

NCT03192397
View directory record Official record
Conditions
Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission, Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission, Chronic Myelomonocytic Leukemia in Remission, Graft Versus Host Disease, Hodgkin Lymphoma, Minimal Residual Disease, Myelodysplastic Syndrome, Myeloproliferative Neoplasm, Non-Hodgkin Lymphoma, Plasma Cell Myeloma, Severe Aplastic Anemia, Waldenstrom Macroglobulinemia
Interventions
Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Melphalan Hydrochloride, Mycophenolate Mofetil, Sirolimus, Total-Body Irradiation
Procedure · Drug · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

NCT05757310
View directory record Official record
Conditions
Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia
Interventions
Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate, Bone Marrow Biopsy, Cyclophosphamide, Haploidentical Hematopoietic Cell Transplantation, Pentostatin, Questionnaire Administration
Biological · Procedure · Drug + 1 more
Lead sponsor
City of Hope Medical Center
Other
Eligibility
Up to 75 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Jan 4, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional Results available

Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant

NCT00343785
View directory record Official record
Conditions
Aplastic Anemia
Interventions
cyclophosphamide, anti-thymocyte globulin, cyclosporine, allogeneic bone marrow transplantation, methotrexate, DNA analysis, flow cytometry, polymorphism analysis, laboratory biomarker analysis
Drug · Biological · Procedure + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 65 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Salt Lake City, Utah • Seattle, Washington • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 12, 2017 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 1 Interventional

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

NCT00692926
View directory record Official record
Conditions
MDS, Anemia, Aplastic, Inborn Errors of Metabolism, Congenital Marrow Failure, Congenital Immunodeficiency Syndrome
Interventions
ALDHbr Umbilical Cord Blood Cells
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 55 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 28, 2017 · Synced Sep 28, 2026, 3:27 AM EDT
Enrolling by invitation No phase listed Observational

Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study

NCT07102849
View directory record Official record
Conditions
Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes, Bone Marrow Diseases
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 120 Years
Enrollment
1,400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 21, 2025 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

NCT00005893
View directory record Official record
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
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 Sep 28, 2026, 3:27 AM EDT
Recruiting No phase listed Observational

Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia

NCT06744283
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 28, 2026, 3:27 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 Sep 28, 2026, 3:27 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 Sep 28, 2026, 3:27 AM EDT
Recruiting Phase 2 Interventional

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Cyclophosphamide, Peripheral Blood Stem Cells
Drug · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 75 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 7, 2026 · Synced Sep 28, 2026, 3:27 AM EDT
Withdrawn Not applicable Interventional

Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

NCT03086252
View directory record Official record
Conditions
Anemia, Aplastic Anemia, Bone Marrow Failure, Hematopoietic and Lymphoid Cell Neoplasm, Leukemia, Myelodysplastic Syndrome
Interventions
Red Blood Cell Transfusions (RBCT)
Procedure
Lead sponsor
Emory University
Other
Eligibility
19 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 25, 2018 · Synced Sep 28, 2026, 3:27 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 Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional Results available

Mismatched Transplantation Using High-dose Post-transplant Cyclophosphamide

NCT01010217
View directory record Official record
Conditions
Blood Stem Cell Transplant Failure, Leukemia, Hematologic Malignancies
Interventions
Cyclophosphamide, Fludarabine, Melphalan, Mesna, Rituximab, Stem Cell Transplantation, Thiotepa, Tacrolimus, Mycofenolate mofetil, G-CSF
Drug · Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Up to 75 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 24, 2020 · Synced Sep 28, 2026, 3:27 AM EDT
Completed Phase 2 Interventional

Low-Dose Danazol for the Treatment of Telomere Related Diseases

NCT03312400
View directory record Official record
Conditions
Telomere Disease
Interventions
Danazol
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 99 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Sep 28, 2026, 3:27 AM EDT