- 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
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Showing 25–48
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- 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
- 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
- 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
- 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
- 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
- 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
- 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