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ClinicalTrials.gov public records Last synced Oct 19, 2026, 9:14 PM EDT

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Showing 1–24 of 301 matching trials from the live ClinicalTrials.gov search.
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
Recruiting No phase listed Observational Accepts healthy volunteers

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT07227155
Conditions
Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
Not listed
Enrollment
375 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2031
U.S. locations
14
States / cities
Duarte, California • Gainesville, Florida • Atlanta, Georgia + 10 more
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
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Severe Aplastic Anemia (SAA)
Interventions
antithymocyte globulin, cyclophosphamide, cyclosporine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
15 Years to 110 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hematologic Malignancies
Interventions
Granulocyte Colony Stimulating Factor
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 22 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2015
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, mesna, methotrexate, methylprednisolone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Fanconi's Anemia
Interventions
amifostine
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
2 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
8 Years to 90 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions
CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
Biological · Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Myelodysplastic Syndromes
Interventions
pacritinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 120 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Aplastic Anemia, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Monoclonal B-Cell Lymphocytosis, Monoclonal Gammopathy of Undetermined Significance, Myelodysplastic Syndrome, Symptomatic COVID-19 Infection Laboratory-Confirmed
Interventions
Best Practice, Ibrutinib
Other · Drug
Lead sponsor
Jennifer Woyach
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
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
Conditions
Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
Interventions
Stem Cell Transplantation
Procedure
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Los Angeles, California
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
Conditions
Congenital Amegakaryocytic Thrombocytopenia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions
anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate, thiotepa, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, in vitro-treated peripheral blood stem cell transplantation, total-body irradiation
Biological · Drug · Procedure + 1 more
Lead sponsor
University of California, San Francisco
Other
Eligibility
1 Year to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
2
States / cities
San Francisco, California • Chapel Hill, North Carolina
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Conditions
Fanconi Anemia
Interventions
metformin HCl
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years to 35 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Leukemia, Inborn Errors of Metabolism, Bone Marrow Failure Syndromes, Immunodeficiencies, Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
Interventions
Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 30 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
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
Completed Phase 1 Interventional Results available

Thrombosomes® in Bleeding Thrombocytopenic Patients

NCT03394755
Conditions
Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
Interventions
Thrombosomes
Biological
Lead sponsor
Cellphire Therapeutics, Inc.
Industry
Eligibility
18 Years to 74 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
6
States / cities
Duarte, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 3 more
Terminated Phase 1Phase 2 Interventional Results available

Stem Cell Transplantation for Fanconi Anemia

NCT00167206
Conditions
Fanconi Anemia
Interventions
Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation, Cyclophosphamide, Fludarabine
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
1 Day to 18 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia, Hodgkin Lymphoma, Indolent Non-Hodgkin Lymphoma, Malignant Neoplasm, Myelodysplastic Syndrome, Myeloproliferative Neoplasm, Plasma Cell Myeloma, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Ring Sideroblasts, Refractory Cytopenia With Multilineage Dysplasia, Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts
Interventions
Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion, Cyclophosphamide, Peripheral Blood Stem Cell Transplantation, Allogeneic Hematopoietic Stem Cell Transplantation, Tacrolimus, Mycophenolate mofetil, Laboratory Biomarker Analysis
Drug · Radiation · Biological + 2 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania