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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Severe Aplastic Anemia (SAA) Clear all
Completed Phase 2 Interventional Results available

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

NCT00004143
View directory record Official record
Conditions
Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions
Campath, Chemo and/or TBI Allo SCT
Drug
Lead sponsor
David Rizzieri, MD
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
2
States / cities
Orlando, Florida • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Dec 4, 2014 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 2 Interventional Results available

Alemtuzumab to Treat Severe Aplastic Anemia

NCT00195624
View directory record Official record
Conditions
Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia, Relapse
Interventions
Alemtuzumab (Campath ), Cyclosporine
Biological · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 110 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 6, 2020 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Not applicable Interventional Results available

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

NCT00244010
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 Jul 21, 2026, 1:18 PM 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 1, 2018 · Synced Jul 21, 2026, 1:18 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia

NCT00590460
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia
Interventions
CAMPATH-1H, Anti-CD45, Fludarabine, Stem cell infusion
Biological · Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2009
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 23, 2018 · Synced Jul 21, 2026, 1:18 PM 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 29, 2008 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 1 Interventional

Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

NCT00054236
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions
anti-thymocyte globulin, filgrastim, cyclophosphamide, fludarabine phosphate, umbilical cord blood transplantation, methylprednisolone
Biological · Drug · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
Up to 120 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 26, 2020 · Synced Jul 21, 2026, 1:18 PM EDT
Recruiting Phase 2 Interventional

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

NCT06412497
View directory record Official record
Conditions
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Rituximab, Rabbit ATG, Cyclophosphamide, Fludarabine, Total Body Irradiation, Cell Infusion, Post-Transplant G-CSF, Tacrolimus, Mycophenolate Mofetil
Drug · Radiation · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 1Phase 2 Interventional

Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation of Patients With Hematological Diseases

NCT00056966
View directory record Official record
Conditions
Hematologic Malignancy
Interventions
ANTI-CD45, CAMPATH-1H, FK506, Fludarabine, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 28, 2012 · Synced Jul 21, 2026, 1:18 PM EDT
Recruiting Phase 2 Interventional

Haploidentical HCT for Severe Aplastic Anemia

NCT04558736
View directory record Official record
Conditions
Aplastic Anemia, Bone Marrow Failure Syndrome
Interventions
Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide, Mesna, G-CSF, Total Lymphoid Irradiation (TLI), CliniMACS, HPC, A Infusion, CD45RA-depleted DLI
Drug · Radiation · Device + 1 more
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Up to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Jul 21, 2026, 1:18 PM EDT
Terminated Phase 1 Interventional Accepts healthy volunteers

Alefacept in Patients With Relapsed/Refractory Aplastic Anemia

NCT01267643
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 Jul 21, 2026, 1:18 PM 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 Jul 21, 2026, 1:18 PM EDT
Completed No phase listed Observational

Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy

NCT04436367
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 Jul 21, 2026, 1:18 PM EDT
Completed Phase 2 Interventional

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

NCT03025698
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Eltrombopag, hATG, CsA
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 18 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
12
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 1 Interventional

Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission

NCT00011830
View directory record Official record
Conditions
Aplastic Anemia
Interventions
G-CSF, Apheresis
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
20 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 3, 2008 · Synced Jul 21, 2026, 1:18 PM EDT
Recruiting No phase listed Observational

HPC Offered for PRESERVE Expansion

NCT07145411
View directory record Official record
Conditions
Bone Marrow Transplant (BMT)
Interventions
Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
Other
Lead sponsor
Ossium Health, Inc.
Industry
Eligibility
12 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Birmingham, Alabama • Detroit, Michigan • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2025 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 2 Interventional Results available

NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia

NCT02833805
View directory record Official record
Conditions
Severe Aplastic Anemia, Aplastic Anemia, Bone Marrow Failure, Immunosuppression
Interventions
Thymoglobulin, Fludarabine, Cyclophosphamide, Total body irradiation, Tacrolimus, Mycophenolate mofetil
Drug · Radiation
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Jul 21, 2026, 1:18 PM EDT
Completed Phase 2 Interventional Results available

Extended Dosing With Eltrombopag for Severe Aplastic Anemia

NCT01891994
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 Jul 21, 2026, 1:18 PM 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 25, 2021 · Synced Jul 21, 2026, 1:18 PM EDT
Active, not recruiting 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 3, 2024 · Synced Jul 21, 2026, 1:18 PM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia

NCT01383434
View directory record Official record
Conditions
Severe Aplastic Anemia
Interventions
Bone Marrow Transplant
Procedure
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
6 Months to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 12, 2016 · Synced Jul 21, 2026, 1:18 PM EDT
Not yet recruiting Phase 2 Interventional

UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia

NCT07001254
View directory record Official record
Conditions
Aplastic Anemia
Interventions
Romiplostim, Immunosuppressive therapy (IST)
Drug
Lead sponsor
Anjali Sharathkumar
Other
Eligibility
2 Years to 21 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Jul 21, 2026, 1:18 PM 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 Jul 21, 2026, 1:18 PM EDT
Completed Not applicable Interventional Results available

Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia

NCT00427336
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 Jul 21, 2026, 1:18 PM EDT