Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
41 studies found for:    "Diamond-Blackfan anemia"
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Rank Status Study
21 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
22 Recruiting Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
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: Drug: fludarabine phosphate;   Drug: melphalan;   Radiation: total-body irradiation;   Drug: tacrolimus;   Drug: mycophenolate mofetil;   Drug: methotrexate;   Other: laboratory biomarker analysis;   Procedure: allogeneic hematopoietic stem cell transplantation;   Procedure: peripheral blood stem cell transplantation
23 Suspended Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
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
Intervention: Procedure: Haploidentical Bone Marrow/Kidney
24 Suspended 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 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
28 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
29 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
30 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
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 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
33 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
34 Recruiting Cancer in Inherited Bone Marrow Failure Syndromes
Conditions: Aplastic Anemia;   Head and Neck Neoplasms;   Bone Marrow Failure
Intervention:
35 Completed Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Conditions: Genetic Disorders;   Sickle Cell Anemia
Intervention: Procedure: Busulfan, Cyclophosphamide, BMD
36 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
37 Recruiting Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT
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 Unknown  Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Conditions: Sickle Cell Disease;   Thalassemia;   Anemia;   Granuloma;   Wiskott-Aldrich Syndrome;   Chediak Higashi Syndrome;   Osteopetrosis;   Neutropenia;   Thrombocytopenia;   Hurler Disease;   Niemann-Pick Disease;   Fucosidosis
Intervention: Procedure: Hematopoietic stem cell transplantation
40 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

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Indicates status has not been verified in more than two years