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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 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
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
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
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
Conditions
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
Interventions
Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
Drug · Biological · Procedure + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
1 Year to 49 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
24
States / cities
Los Angeles, California • San Diego, California • San Francisco, California + 20 more
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
Terminated Not applicable Interventional Results available

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

NCT01319851
Conditions
Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Schwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Fanconi Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia
Interventions
Alefacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Atlanta, Georgia
Recruiting No phase listed Observational Accepts healthy volunteers

Cancer in Inherited Bone Marrow Failure Syndromes

NCT00027274
Conditions
Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome, Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Day to 100 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
2
States / cities
Bethesda, Maryland • Rockville, Maryland
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Bone marrow transplantation, Mesenchymal Stromal Cells
Procedure · Biological
Lead sponsor
Stanford University
Other
Eligibility
1 Year to 25 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
2
States / cities
Birmingham, Alabama • Minneapolis, Minnesota
Conditions
Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
175 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
6
States / cities
Oakland, California • Stanford, California • Arlington Heights, Illinois + 3 more
Conditions
Purpura, Schoenlein-Henoch, Graft Versus Host Disease, Anemia, Hemolytic, Autoimmune, Rheumatoid Arthritis, Churg-Strauss Syndrome, Hypersensitivity Vasculitis, Wegener's Granulomatosis, Systemic Lupus Erythematosus, Giant Cell Arteritis, Pure Red Cell Aplasia, Juvenile Rheumatoid Arthritis, Polyarteritis Nodosa, Autoimmune Thrombocytopenic Purpura, Takayasu Arteritis
Interventions
anti-thymocyte globulin, cyclophosphamide, cyclosporine, filgrastim, methylprednisolone, prednisone, Autologous Peripheral Blood Stem Cell Transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
1 Year to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Recruiting No phase listed Observational

Diamond Blackfan Anemia Registry (DBAR)

NCT00106015
Conditions
Anemia, Blood Disease
Interventions
Not listed
Lead sponsor
Northwell Health
Other
Eligibility
Not listed
Enrollment
900 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2030
U.S. locations
1
States / cities
New Hyde Park, New York
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
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
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
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Bone Marrow Failure Syndromes

NCT00176878
Conditions
Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Interventions
Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation, Busulfan, anti-thymocyte globulin
Procedure · Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Terminated Phase 1Phase 2 Interventional Results available

The Use of Trifluoperazine in Transfusion Dependent DBA

NCT03966053
Conditions
Diamond Blackfan Anemia, Pure Red Cell Aplasia
Interventions
Trifluoperazine
Drug
Lead sponsor
Adrianna Vlachos, MD
Other
Eligibility
18 Years to 65 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Boston, Massachusetts • Manhasset, New York
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
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
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Not listed
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
16 Years and older
Enrollment
423 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
6
States / cities
Oakland, California • Augusta, Georgia • Chicago, Illinois + 3 more
Conditions
Chronic Kidney Disease, Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkin's Lymphoma (NHL), Hodgkin Disease, Multiple Myeloma, Myelodysplastic Syndrome (MDS), Aplastic Anemia, AL Amyloidosis, Diamond Blackfan Anemia, Myelofibrosis, Myeloproliferative Disease, Sickle Cell Anemia, Autoimmune Diseases, Thalassemia
Interventions
Haploidentical Bone Marrow/Kidney
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts