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ClinicalTrials.gov public records Last synced Oct 5, 2026, 6:19 PM EDT

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Showing 25–38 of 38 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Severe Aplastic Anemia, Refractory Clear all
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 Sep 22, 2026 · Synced Oct 5, 2026, 6:19 PM EDT
Completed Phase 2 Interventional Results available

Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome

NCT00604201
View directory record Official record
Conditions
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA), Severe Aplastic Anemia (SAA)
Interventions
Umbilical Cord Blood
Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 5, 2022 · Synced Oct 5, 2026, 6:19 PM EDT
Completed Phase 2 Interventional Results available

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 Aug 10, 2026 · Synced Oct 5, 2026, 6:19 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 25, 2023 · Synced Oct 5, 2026, 6:19 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 27, 2020 · Synced Oct 5, 2026, 6:19 PM EDT
Completed Not applicable Interventional Results available

Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies

NCT01499147
View directory record Official record
Conditions
Acute Myeloid Leukemia, Acute Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma, Hodgkin's Disease, Multiple Myeloma, Lymphocytic Leukemia, Myeloproliferative Disorder, Polycythemia Vera, Myelofibrosis, Aplastic Anemia
Interventions
fludarabine/busulfan, fludarabine/ melphalan, ATG
Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
10 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2013
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 8, 2018 · Synced Oct 5, 2026, 6:19 PM EDT
Completed Phase 2 Interventional Results available

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

NCT01529827
View directory record Official record
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Grade III Lymphomatoid Granulomatosis, Adult Nasal Type Extranodal NK/T-cell Lymphoma, Anaplastic Large Cell Lymphoma, Angioimmunoblastic T-cell Lymphoma, Aplastic Anemia, Burkitt Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Diffuse Large Cell Lymphoma, Childhood Grade III Lymphomatoid Granulomatosis, Childhood Immunoblastic Large Cell Lymphoma, Childhood Myelodysplastic Syndromes, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Juvenile Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Paroxysmal Nocturnal Hemoglobinuria, Peripheral T-cell Lymphoma, Polycythemia Vera, Post-transplant Lymphoproliferative Disorder, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Adult T-cell Leukemia/Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Refractory Multiple Myeloma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Secondary Myelofibrosis, Severe Combined Immunodeficiency, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Splenic Marginal Zone Lymphoma, T-cell Large Granular Lymphocyte Leukemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Radiation · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
3 Years to 75 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Sep 24, 2019 · Synced Oct 5, 2026, 6:19 PM 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 5, 2026, 6:19 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia

NCT01187017
View directory record Official record
Conditions
Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
Interventions
Cyclophosphamide, Fludarabine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2021 · Synced Oct 5, 2026, 6:19 PM EDT
Recruiting No phase listed Observational

Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer

NCT01137643
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions
biologic sample preservation procedure, cytology specimen collection procedure
Other
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
15,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2040
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 27, 2025 · Synced Oct 5, 2026, 6:19 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 5, 2026 · Synced Oct 5, 2026, 6:19 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 19, 2025 · Synced Oct 5, 2026, 6:19 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 13, 2016 · Synced Oct 5, 2026, 6:19 PM EDT
Recruiting Phase 2 Interventional

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

NCT02143830
View directory record Official record
Conditions
Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
Interventions
Busulfan, Cyclophosphamide, Fludarabine, rabbit ATG, G-CSF, Peripheral blood stem cell
Drug · Biological
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
3 Months and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
3
States / cities
New York, New York • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 5, 2026, 6:19 PM EDT