1. Completed: Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

Condition: Diamond Blackfan Anemia

Interventions: Drug: G-CSF; Procedure: Leukapheresis

 

2. Recruiting: Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Conditions: Anemia, Aplastic; Red-Cell Aplasia, Pure; Anemia, Diamond-Blackfan

Intervention: Drug: Rituximab (Rituxan)

 

3. Completed: Medical Treatment for Diamond Blackfan Anemia

Conditions: Fanconi's Anemia; Hematologic Disease

Interventions: Drug: Antithymocyte globulin; Drug: Cyclosporine

 

4. Recruiting: Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

Conditions: Congenital Hemolytic Anemia; Diamond-Blackfan Anemia

Interventions: Procedure: Radiotherapy; Drug: Alemtuzumab (Campath® (Registered Trademark)); Drug: Sirolimus (Rapamune® (Registered Trademark))

 

5. Recruiting: A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

Conditions: Aplastic Anemia; Pure Red Cell Aplasia; Diamond Blackfan Anemia

Interventions: Drug: Daclizumab; Drug: Daclizumab (Zenapax)

 

6. Completed: Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

Conditions: Genetic Disorders; Sickle Cell Anemia

Interventions: Procedure: Busulfan, Cyclophosphamide, BMD

 

7. Active, not recruiting: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Conditions: Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis Interventions: Drug: Deferasirox

 

8. Recruiting: Diamond Blackfan Anemia Registry (DBAR)

Conditions: Anemia; Blood Disease

 

9. Recruiting: Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Conditions: Anemia, Aplastic; Amegakaryocytic Thrombocytopenia; Diamond-Blackfan Anemia; Kostmann Syndrome

Interventions: Procedure: Allogeneic stem cell transplant

 

10. Completed: Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

Conditions: Cancer-Related Problem/Condition; Congenital Amegakaryocytic Thrombocytopenia; Diamond-Blackfan Anemia; Fanconi Anemia; Leukemia; Severe Congenital Neutropenia

Interventions: Drug: anti-thymocyte globulin; Drug: busulfan; Drug: fludarabine phosphate; Procedure: allogeneic bone marrow transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation

 

11. Active, not recruiting: Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Conditions: Chronic Myeloproliferative Disorders; Diamond-Blackfan Anemia; Fanconi Anemia; Graft Versus Host Disease; Leukemia; Lymphoma; Multiple Myeloma and Plasma Cell Neoplasm; Myelodysplastic Syndromes; Myelodysplastic/Myeloproliferative Diseases

Interventions: Drug: anti-thymocyte globulin; Drug: busulfan; Drug: cyclophosphamide; Drug: cyclosporine; Drug: filgrastim; Drug: melphalan; Drug: methylprednisolone; Drug: mycophenolate mofetil; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation

 

12. Recruiting: Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

Conditions: Congenital Amegakaryocytic Thrombocytopenia; Diamond-Blackfan Anemia; Leukemia; Myelodysplastic Syndromes; Severe Congenital Neutropenia

Interventions: Drug: alemtuzumab; Drug: busulfan; Drug: cyclosporine; Drug: fludarabine phosphate; Drug: methotrexate; Drug: methylprednisolone; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: umbilical cord blood transplantation

 

13. Recruiting: Stem Cell Transplant for Bone Marrow Failures

Conditions: Diamond-Blackfan Anemia; Kostmann's Neutropenia; Shwachman-Diamond Syndrome

Interventions: Procedure: Stem cell transplant; Drug: Busulfan, fludarabine and anti-thymocyte globulin (ATG); Procedure: Total lymphoid irradiation

 

14. Recruiting: Stem Cell Transplant for Hemoglobinopathy

Conditions: Sickle Cell Disease; Thalassemia; Severe Congenital Neutropenia; Diamond-Blackfan Anemia; Shwachman-Diamond Syndrome

Interventions: Drug: Cyclophosphamide, Fludarabine, Campath; Drug: Busulfan, Cyclophosphamide, ATG

 

15. Recruiting: Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload

Conditions: Transfusional Iron Overload in the Heart; Transfusional Hemosiderosis

Interventions: Drug: Deferasirox; Drug: Deferoxamine

 

16. Recruiting: 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

Interventions: Other: Unrelated Umbilical Cord Blood Transplant

 

17. Recruiting: Genetic Study of Cancer Risk and Gene Identification in Patients With Inherited Bone Marrow Disorders and Their Families

Conditions: Cancer

Interventions: Procedure: biologic sample preservation procedure; Procedure: comparative genomic hybridization; Procedure: cytogenetic analysis; Procedure: gene expression profiling; Procedure: medical chart review; Procedure: questionnaire administration

 

18. Active, not recruiting: A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-Dependent Iron Overload

Conditions: Transfusion-Dependent Iron Overload

Interventions: Drug: Deferasirox

 

19. Active, not recruiting: Efficacy and Safety of Deferasirox in Patients With Transfusion-Dependent Iron Overload

Conditions: Transfusion-Dependent Iron Overload

Interventions: Drug: Deferasirox

 

20. Recruiting: ICL670 in Patients With Chronic Anemia and Transfusional Hemosiderosis

Conditions: Anemia; Hemosiderosis

Interventions: Drug: Deferasirox (ICL670)

 

21. Completed: Study of Deferasirox in Iron Overload From Beta-Thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Conditions: Beta-Thalassemia; Myelodysplastic Syndromes; Fanconi Syndrome; Anemia, Diamond-Blackfan; Anemia, Aplastic

Interventions: Drug: Deferasirox

 

22. Recruiting: Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Conditions: Red Blood Cell Defects; Sickle Cell Disease; Thalassemia; Blackfan-Diamond Anemia; Leukocyte Defects and Immune Deficiencies; Hereditary Lymphohistiocytosis; Chronic Granulomatous Disease; Wiskott-Aldrich Syndrome; Chediak Higashi Syndrome; CD40 Ligand Deficiency; Hyper IgM Syndrome; Leucocytes Adhesion Defect Type 1; Osteopetrosis; Congenital Neutropenia; X-Linked Lymphoproliferative Disease; Platelets Defects; Congenital Amegakaryocytic Thrombocytopenia; Metabolic and Storage Disorders; Hurler Disease; Leukodystrophies; Niemann-Pick Disease; Fucosidosis; Stem Cell Defects; Reticular Agenesis

Interventions: Procedure: Hematopoietic stem cell transplantation