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Showing 1–24 of 84 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Aplastic Anemia (SAA) Clear all
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, 9:35 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, 9:35 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, 9:35 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, 9:35 AM EDT
Completed Phase 2 Interventional Results available

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

NCT01231841
View directory record Official record
Conditions
Aplastic Anemia
Interventions
cyclosporine, anti-thymocyte globulin
Drug · Biological
Lead sponsor
The Cleveland Clinic
Other
Eligibility
12 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 21, 2023 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)

NCT00326417
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 Jul 21, 2026, 9:35 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 Jul 21, 2026, 9:35 AM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jul 21, 2026, 9:35 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 Jul 1, 2026 · Synced Jul 21, 2026, 9:35 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 Jul 21, 2026, 9:35 AM EDT
Completed No phase listed Observational

Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy

NCT04436380
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
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 15, 2023 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Not applicable Interventional

Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

NCT02845596
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 Jul 21, 2026, 9:35 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 7, 2025 · Synced Jul 21, 2026, 9:35 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 4, 2017 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Phase 2 Interventional

Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions

NCT00003336
View directory record Official record
Conditions
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, melphalan, methylprednisolone, bone marrow ablation with stem cell support, umbilical cord blood transplantation, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2006
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 10, 2010 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
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 Jul 21, 2026, 9:35 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 7, 2017 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Not applicable Interventional

Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia

NCT00186797
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Allogeneic stem cell transplant, Fludarabine, Cyclophosphamide
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 8, 2010 · Synced Jul 21, 2026, 9:35 AM EDT
Active, not recruiting Phase 3 Interventional

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

NCT05600426
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 Jul 14, 2026 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia

NCT01193283
View directory record Official record
Conditions
Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
Interventions
Cyclophosphamide, Cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 21, 2017 · Synced Jul 21, 2026, 9:35 AM EDT
Terminated Phase 1 Interventional

REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy

NCT04409080
View directory record Official record
Conditions
Severe Aplastic Anemia (SAA)
Interventions
REGN7257
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Cleveland, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Jul 21, 2026, 9:35 AM EDT
Completed Phase 2 Interventional

Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

NCT00004464
View directory record Official record
Conditions
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Up to 70 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2008
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 17, 2019 · Synced Jul 21, 2026, 9:35 AM EDT
Available No phase listed Expanded access

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

NCT01881334
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 Jul 21, 2026, 9:35 AM EDT
Recruiting No phase listed Observational

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

NCT01351545
View directory record Official record
Conditions
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
Interventions
A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Drug
Lead sponsor
Center for International Blood and Marrow Transplant Research
Network
Eligibility
Not listed
Enrollment
99,999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2041
U.S. locations
142
States / cities
Birmingham, Alabama • Gilbert, Arizona • Phoenix, Arizona + 90 more
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 21, 2026, 9:35 AM EDT