Clinical Trials

Listed below are clinical trials which have been added or updated in the last 90 days. If you would like to see the full list of clinical trials please click here.

  • Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
    Condition:   Anemia, Diamond-Blackfan
    Intervention:   Drug: Eltrombopag
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    Recruiting
  • T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
    Conditions:   Sickle Cell Anemia;   Beta-thalassemia Major;   Diamond-blackfan Anemia
    Interventions:   Biological: CD3/CD19 depleted leukocytes;   Biological: CD45RA depleted leukocytes;   Drug: Hydroxyurea;   Drug: Rituximab;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Thiotepa
    Sponsor:   Paul Szabolcs
    Recruiting
  • Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
    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:   Drug: Thiotepa–single daily dose;   Drug: Thiotepa–escalated dose
    Sponsors:   University of Florida;   Live Like Bella Pediatric Cancer Research
    Recruiting
  • Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
    Conditions:   Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;   Acute Leukemia in Remission;   Acute Lymphoblastic Leukemia;   Acute Myeloid Leukemia;   Acute Myeloid Leukemia With FLT3/ITD Mutation;   Acute Myeloid Leukemia With Gene Mutations;   Aplastic Anemia;   B-Cell Non-Hodgkin Lymphoma;   CD40 Ligand Deficiency;   Chronic Granulomatous Disease;   Chronic Leukemia in Remission;   Chronic Lymphocytic Leukemia;   Chronic Myelogenous Leukemia, BCR-ABL1 Positive;   Chronic Myelomonocytic Leukemia;   Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;   Congenital Amegakaryocytic Thrombocytopenia;   Congenital Neutropenia;   Congenital Pure Red Cell Aplasia;   Glanzmann Thrombasthenia;   Immunodeficiency Syndrome;   Myelodysplastic Syndrome;   Myelofibrosis;   Myeloproliferative Neoplasm;   Paroxysmal Nocturnal Hemoglobinuria;   Plasma Cell Myeloma;   Polycythemia Vera;   Recurrent Non-Hodgkin Lymphoma;   Refractory Non-Hodgkin Lymphoma;   Secondary Acute Myeloid Leukemia;   Secondary Myelodysplastic Syndrome;   Severe Aplastic Anemia;   Shwachman-Diamond Syndrome;   Sickle Cell Disease;   T-Cell Non-Hodgkin Lymphoma;   Thalassemia;   Waldenstrom Macroglobulinemia;   Wiskott-Aldrich Syndrome
    Interventions:   Drug: Cyclophosphamide;   Drug: Fludarabine Phosphate;   Other: Laboratory Biomarker Analysis;   Procedure: Peripheral Blood Stem Cell Transplantation;   Radiation: Total-Body Irradiation
    Sponsor:   Roswell Park Cancer Institute
    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 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
    Intervention:  
    Sponsor:   St. Jude Children’s Research Hospital
    Recruiting
  • Fludarabine Based RIC for Bone Marrow Failure Syndromes
    Condition:   Bone Marrow Failure Syndromes
    Interventions:   Other: MRD-BMT with Fludarabine-based RIC for Acquired AA;   Other: MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia;   Other: MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia
    Sponsor:   Children’s Hospital of Philadelphia
    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;   Myelodysplastic Syndromes;   Myeloproliferative Diseases
    Intervention:  
    Sponsors:   St. Jude Children’s Research Hospital;   Boston Children’s Hospital;   University of Memphis;   Monroe Carell Jr. Children’s Hospital at Vanderbilt;   Baylor College of Medicine;   Children’s Hospital of Philadelphia
    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
    Sponsor:   Masonic Cancer Center, University of Minnesota
    Recruiting
  • Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
    Conditions:   Primary Immunodeficiency (PID);   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Inflammatory Conditions
    Interventions:   Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
    Sponsor:   Paul Szabolcs
    Recruiting
  • Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
    Conditions:   Congenital Hemolytic Anemia;   Sickle Cell Disease
    Interventions:   Procedure: Peripheral blood hematopoietic progenitor cell (PBPC) transplant;   Drug: Alemtuzumab;   Procedure: Peripheral blood hematopoietic progenitor cell Apherisis;   Drug: Sirolimus
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    Recruiting
  • Cancer in Inherited Bone Marrow Failure Syndromes
    Conditions:   Diamond Blackfan Anemia;   Dyskeratosis Congenita;   Fanconi Anemia;   Shwachman Diamond Syndrome;   Inherited Bone Marrow Failure Syndrome, Aplastic Anemia
    Intervention:  
    Sponsor:   National Cancer Institute (NCI)
    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
    Intervention:   Drug: Daclizumab
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    Terminated