41 studies found for:    "Diamond-Blackfan anemia"
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21 Terminated Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Conditions: Sickle Cell Disease;   Thalassemia;   Diamond-Blackfan Anemia
Interventions: Procedure: Bone marrow transplantation;   Biological: Mesenchymal Stromal Cells
22 Recruiting Human Placental-Derived Stem Cell Transplantation
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
Intervention: Drug: Human Placental Derived Stem Cell
23 Recruiting Stem Cell Transplant for Hemoglobinopathy
Conditions: Sickle Cell Disease;   Thalassemia;   Severe Congenital Neutropenia;   Diamond-Blackfan Anemia;   Shwachman-Diamond Syndrome
Interventions: Drug: Busulfan, Fludarabine, ATG, TLI;   Drug: Busulfan, Cyclophosphamide, ATG, GCSF;   Drug: Campath, Fludarabine, Cyclophosphamide;   Radiation: Total Body Irradiation;   Procedure: Stem cell infusion
24 Active, not recruiting Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
Conditions: Anemia, Sickle Cell;   Complex and Transfusion-dependent Hemoglobinopathies;   Thalassemia;   Alpha or Beta Thalassemia Major;   Diamond-Blackfan Anemia;   Bone Marrow Failure Syndromes Characterized by Severe Chronic Anemia
Intervention: Device: Enriched Hematopoetic Stem Cell Transplantation
25 Active, not recruiting Multi-Center Study of Iron Overload: Pilot Study
Conditions: Sickle Cell Disease;   Thalassemia;   Diamond-Blackfan Anemia
Intervention:
26 Recruiting Diamond Blackfan Anemia Registry (DBAR)
Conditions: Anemia;   Blood Disease
Intervention:
27 Completed Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
Conditions: Haemoglobinopathies,;   Myelodysplastic Syndromes,;   Other Inherited or Acquired Anaemia (e.g. MPD, Diamond-blackfan Anaemia and Other Rare Anaemias),;   Transfusional Iron Overload
Intervention: Drug: deferasirox
28 Unknown  Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
Conditions: Anemia;   Leukemia;   Leukemia, Myelocytic, Acute;   Dysmyelopoietic Syndromes
Intervention: Drug: Lenalidomide
29 Completed
Has Results
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
Conditions: Transfusional Iron Overload;   Transfusional Hemosiderosis
Interventions: Drug: Core Study: Deferasirox;   Drug: Core Study: Deferoxamine;   Drug: Extension: deferoxamine to deferasirox;   Drug: Extension: deferasirox to deferoxamine;   Drug: Deferasirox;   Drug: Deferoxamine
30 Completed Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Conditions: Genetic Disorders;   Sickle Cell Anemia
Intervention: Procedure: Busulfan, Cyclophosphamide, BMD
31 Recruiting Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Condition: Nonmalignant Neoplasm
Interventions: Drug: treosulfan;   Drug: fludarabine phosphate;   Biological: anti-thymocyte globulin;   Radiation: total-body irradiation;   Procedure: allogeneic bone marrow transplantation;   Procedure: peripheral blood stem cell transplantation;   Procedure: umbilical cord blood transplantation;   Drug: tacrolimus;   Drug: methotrexate;   Drug: cyclosporine;   Drug: mycophenolate mofetil;   Other: laboratory biomarker analysis
32 Not yet recruiting Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Conditions: Primary Immune Deficiency Disorders;   Hemophagocytic Lymphohistiocytosis;   Inherited Bone Marrow Failure Syndrome;   Hemoglobinopathies;   Metabolic Disorders
Intervention: Biological: BPX-501 and AP1903
33 Recruiting CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
Conditions: Chronic Myeloid Leukemia (CML);   Acute Myelogenous Leukemia (AML);;   Myelodysplastic Syndrome (MDS);;   Juvenile Myelomonocytic Leukemia (JMML);;   Acute Lymphoblastic Leukemia (ALL);;   Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions: Device: CliniMACS CD34+ Reagent System;   Drug: Thiotepa;   Drug: Cyclophosphamide;   Drug: Alemtuzumab;   Drug: Tacrolimus;   Drug: Melphalan;   Drug: Busulfan;   Drug: Fludarabine
34 Recruiting Cancer in Inherited Bone Marrow Failure Syndromes
Conditions: Fanconi's Anemia;   Anemia, Diamond Blackfan;   Dyskeratosis Congenital;   Thrombocytopenia;   Neutropenia
Intervention:
35 Completed A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
Condition: Transfusion-dependent Iron Overload
Intervention: Drug: Deferasirox
36 Completed
Has Results
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Conditions: Non Malignant Disorders;   Immunodeficiencies;   Congenital Marrow Failures;   Hemoglobinopathies;   Inborn Errors of Metabolism;   Sickle Cell;   Thalassemia;   Lysosomal Storage Disease
Interventions: Biological: Unrelated Umbilical Cord Blood Transplant;   Drug: Reduced Intensity Conditioning
37 Recruiting Reduced Intensity Conditioning in Patients Aged ≤30 With Non-Malignant Disorders Undergoing Cord Blood Transplantation
Conditions: Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
Interventions: Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
38 Completed A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
Condition: Transfusional Iron Overload
Intervention: Drug: ICL670
39 Completed Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
Conditions: Neutropenia;   Sickle Cell Anemia;   Thalassemia Major;   Red-Cell Aplasia, Pure
Interventions: Drug: anti-thymocyte globulin;   Drug: busulfan;   Drug: cyclophosphamide;   Procedure: Bone marrow transplantation
40 Completed Stem Cell Transplantation (SCT) for Genetic Diseases
Conditions: Thrombocytopenia;   Metachromatic Leukodystrophy;   Fanconi's Anemia;   Thalassemia Major;   Pure Red-Cell Aplasia;   Inborn Errors of Metabolism
Intervention: Procedure: Stem Cell Transplantation

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Indicates status has not been verified in more than two years