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45 studies found for:    "Diamond-Blackfan anemia"
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Rank Status Study
21 Recruiting CD34+ Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation for Non-Malignant Disease
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
Intervention: Biological: CD34 Stem Cell Selection Therapy
22 Suspended
Has Results
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
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
Intervention: Drug: Alefacept
23 Recruiting Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
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: Drug: Reduced Toxicity Ablative Regimen;   Drug: Reduced Intensity Preparative Regimen;   Drug: Myeloablative Preparative Regimen
24 Suspended Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
Conditions: Anemia, Sickle Cell;   Complex and Transfusion-dependent Hemoglobinopathies;   Thalassemia;   Diamond-Blackfan Anemia;   Bone Marrow Failure Syndromes;   Alpha-Thalassemia;   Beta-Thalassemia
Intervention: Biological: Enriched Hematopoetic Stem Cell Infusion
25 Completed Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
Conditions: Hemoglobinopathies;   Myelodysplastic Syndromes;   Other Inherited or Acquired Anaemia;   MPD Syndrome;   Diamond-Blackfan Anemia;   Other Rare Anaemias;   Transfusional Iron Overload
Intervention: Drug: deferasirox
26 Recruiting Familial Investigations of Childhood Cancer Predisposition
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 Leukaemia;   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
Intervention:
27 Recruiting Investigation of the Genetics of Hematologic Diseases
Conditions: Bone Marrow Failure Syndromes;   Erythrocyte Disorder;   Leukocyte Disorder;   Hemostasis;   Blood Coagulation Disorder;   Sickle Cell Disease;   Dyskeratosis Congenita;   Diamond-Blackfan Anemia;   Congenital Thrombocytopenia;   Severe Congenital Neutropenia;   Fanconi Anemia
Intervention:
28 Terminated
Has Results
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
Conditions: Stem Cell Transplantation;   Bone Marrow Transplantation;   Peripheral Blood Stem Cell Transplantation;   Allogeneic Transplantation;   Genetic Diseases;   Thalassemia;   Pediatrics;   Diamond-Blackfan Anemia;   Combined Immune Deficiency;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   X-linked Lymphoproliferative Disease;   Metabolic Diseases
Interventions: Drug: Cyclophosphamide Dose Level 1;   Drug: Cyclophosphamide Dose Level 2;   Drug: Cyclophosphamide Dose Level 3;   Drug: Cyclophosphamide Dose Level 4
29 Recruiting BMT Abatacept for Non-Malignant Diseases
Conditions: Hurler Syndrome;   Fanconi Anemia;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Shwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia;   Thalassemia Major;   Hemophagocytic Lymphohistiocytosis;   Sickle Cell Disease
Intervention: Drug: Abatacept
30 Recruiting 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
31 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
32 Active, not 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
33 Completed Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Conditions: Genetic Disorders;   Sickle Cell Anemia
Intervention: Procedure: Busulfan, Cyclophosphamide, BMD
34 Recruiting Cancer in Inherited Bone Marrow Failure Syndromes
Conditions: Aplastic Anemia;   Head and Neck Neoplasms;   Bone Marrow Failure
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 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
37 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;   Drug: Methylprednisolone
38 Recruiting Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Condition: Non-Malignant
Interventions: Procedure: Allogeneic Bone Marrow Transplantation;   Biological: Anti-Thymocyte Globulin;   Drug: Cyclosporine;   Drug: Fludarabine Phosphate;   Other: Laboratory Biomarker Analysis;   Drug: Methotrexate;   Drug: Mycophenolate Mofetil;   Procedure: Peripheral Blood Stem Cell Transplantation;   Drug: Tacrolimus;   Radiation: Total-Body Irradiation;   Drug: Treosulfan;   Procedure: Umbilical Cord Blood Transplantation
39 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
40 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

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Study has passed its completion date and status has not been verified in more than two years.