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ClinicalTrials.gov public records Last synced May 21, 2026, 11:11 PM EDT

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Showing 1–24 of 83 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Aplastic Anemia (SAA) Clear all
Available No phase listed Expanded access
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Chronic Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplasia, Acute Lymphocytic Leukemia, Severe Aplastic Anemia, Non-Hodgkin's Lymphoma, Lymphoproliferative Disease, Multiple Myeloma, Advanced Myeloproliferative Disease
Interventions
Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSF
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
15 Years to 61 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 25, 2009 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 11:11 PM EDT
Active, not recruiting Not applicable Interventional Results available
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia, Hemophagocytic Lymphohistiocytoses, Primary Immunodeficiency Diseases, Hemoglobinopathies, Severe Aplastic Anemia, Cytopenia, Bone Marrow Failure Syndrome, Severe Chronic Active Epstein-Barr Virus Infection
Interventions
CliniMACS
Device
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced May 21, 2026, 11:11 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions
Allogeneic stem cell transplant
Device
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 29, 2017 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 24, 2023 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 1Phase 2 Interventional
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 29, 2008 · Synced May 21, 2026, 11:11 PM EDT
Terminated Phase 2 Interventional Results available
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 3, 2025 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Anemia, Aplastic
Interventions
Fludarabine, Cyclophosphamide 150mg, Cyclophosphamide 100mg, Cyclophosphamide 50mg
Drug
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Up to 65 Years
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
22
States / cities
Phoenix, Arizona • Duarte, California • Los Angeles, California + 17 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced May 21, 2026, 11:11 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Fludarabine, Cyclophosphamide, Antithymocyte Globulin
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Up to 70 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Oct 26, 2011 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Aplastic Anemia, Bone Marrow Failure Syndromes
Interventions
Bone marrow transplant, Thymoglobulin, Fludarabine, Cyclophosphamide, TBI, Mesna, Tacrolimus, Mycophenolic acid mofetil
Procedure · Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 73 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
2
States / cities
Baltimore, Maryland • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 9, 2023 · Synced May 21, 2026, 11:11 PM EDT
Completed Phase 2 Interventional Results available
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 May 21, 2026, 11:11 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 99 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced May 21, 2026, 11:11 PM EDT
Recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 99 Years
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced May 21, 2026, 11:11 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers
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 May 21, 2026, 11:11 PM EDT
Active, not recruiting Phase 3 Interventional
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 – 2029
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 44 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 21, 2026, 11:11 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6, Extension Cohort
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 95 Years
Enrollment
207 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced May 21, 2026, 11:11 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
cyclosporine, Matched Unrelated Donor Hematopoietic 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
Michael Pulsipher
Other
Eligibility
Up to 25 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
13
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated May 10, 2025 · Synced May 21, 2026, 11:11 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Alefacept
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 25, 2018 · Synced May 21, 2026, 11:11 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced May 21, 2026, 11:11 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available
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 May 21, 2026, 11:11 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 25, 2019 · Synced May 21, 2026, 11:11 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Sirolimus
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced May 21, 2026, 11:11 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL, Hypoplastic MDS
Interventions
Ruxolitinib
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced May 21, 2026, 11:11 PM EDT