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ClinicalTrials.gov public records Last synced Jul 24, 2026, 12:48 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Conditions
Bone Marrow Failure Syndromes, Immunodeficiencies, Immune Dysregulation Syndromes
Interventions
CliniMACs device
Device
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 22 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
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
Conditions
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Aplastic Anemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Hematopoietic and Lymphoid Cell Neoplasm, Hodgkin Lymphoma, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Non-Hodgkin Lymphoma, Plasma Cell Myeloma
Interventions
Health Promotion and Education, Medical Device Usage and Evaluation, Quality-of-Life Assessment, Questionnaire Administration
Other
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
60 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Fanconi Anemia
Interventions
AMD3100, AmCell CliniMACs
Drug · Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Year to 30 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Conditions
Bone Marrow Disease
Interventions
CK0801
Biological
Lead sponsor
Cellenkos, Inc.
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
Santa Monica, California • Houston, Texas
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Melphalan
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
San Francisco, California
Conditions
Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
Interventions
Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi)
Drug
Lead sponsor
New York Medical College
Other
Eligibility
Up to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Valhalla, New York
Conditions
Blasts 10-19 Percent of Bone Marrow Nucleated Cells, Blasts More Than 5 Percent of Bone Marrow Nucleated Cells, High Risk Chronic Myelomonocytic Leukemia, Recurrent Chronic Myelomonocytic Leukemia, Recurrent High Risk Myelodysplastic Syndrome, Refractory Chronic Myelomonocytic Leukemia, Refractory High Risk Myelodysplastic Syndrome
Interventions
Liposome-encapsulated Daunorubicin-Cytarabine
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Houston, Texas
Completed Phase 1Phase 2 Interventional Results available

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

NCT04269889
Conditions
Anemia, Diamond-Blackfan
Interventions
Eltrombopag
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Leukemia, Non Hodgkin's Lymphoma, Aplastic Anemia
Interventions
Megadose T-cell Depleted HLA-nonidentical Blood Progenitor Cell Transplantation
Procedure
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2004
U.S. locations
1
States / cities
Houston, Texas
Conditions
Myelodysplastic Syndrome
Interventions
Tosedostat
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
New York, New York
Conditions
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia, Adult Acute Monocytic Leukemia, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11, Adult Acute Myeloid Leukemia With Maturation, Adult Acute Myeloid Leukemia With Minimal Differentiation, Adult Acute Myeloid Leukemia With t(16;16)(p13.1;q22); CBFB-MYH11, Adult Acute Myeloid Leukemia With t(8;21); (q22; q22.1); RUNX1-RUNX1T1, Adult Acute Myeloid Leukemia With t(9;11)(p22.3;q23.3); MLLT3-KMT2A, Adult Acute Myeloid Leukemia Without Maturation, Adult Acute Myelomonocytic Leukemia, Adult Erythroleukemia, Adult Pure Erythroid Leukemia, Alkylating Agent-Related Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts, Recurrent Adult Acute Myeloid Leukemia
Interventions
Clinical Observation, Tipifarnib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2015
U.S. locations
339
States / cities
Birmingham, Alabama • Anchorage, Alaska • Scottsdale, Arizona + 228 more
Conditions
Graft Versus Host Disease, Aplastic Anemia
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, methylprednisolone, Allogeneic Bone Marrow Transplantation
Drug · Procedure
Lead sponsor
Northwestern Memorial Hospital
Other
Eligibility
15 Years to 55 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Chicago, Illinois
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
Completed No phase listed Observational Accepts healthy volunteers

Protection Against Benzene Toxicity

NCT00011453
Conditions
Leukemia, Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Institute of Environmental Health Sciences (NIEHS)
NIH
Eligibility
21 Years to 65 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2001
U.S. locations
1
States / cities
Denver, Colorado
Conditions
Aplastic Anemia, de Novo Myelodysplastic Syndrome, Myelodysplastic Syndrome, Previously Treated Myelodysplastic Syndrome
Interventions
Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, Methylprednisolone, Pegfilgrastim
Biological · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2029
U.S. locations
1
States / cities
Houston, Texas
Conditions
Telomere Disease
Interventions
Danazol
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 99 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Aplastic Anemia, Pancytopenia
Interventions
Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF)
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Trauma Injury, Trauma, Critical Illness, Microbiome, Chronic Anemia, Acute Blood Loss Anemia
Interventions
Data and tissue collection
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 100 Years
Enrollment
275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
3
States / cities
Gainesville, Florida
Conditions
Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes, Bone Marrow Diseases
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 120 Years
Enrollment
1,400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Fanconi Anemia
Interventions
JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant, Rabbit Anti-Thymoglobulin (rATG), Cyclophosphamide, Fludarabine, Rituximab
Drug · Device · Biological
Lead sponsor
Porteus, Matthew, MD
Other
Eligibility
2 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Stanford, California
Conditions
Infantile Malignant Osteopetrosis
Interventions
RP-L401
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Month and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Los Angeles, California