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.

  • Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
    Conditions:   Primary Immunodeficiency (PID);   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Inflammatory Conditions
    Intervention:   Drug: data collection
    Sponsor:   Paul Szabolcs
    Recruiting
  • Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
    Condition:   Anemia, Diamond-Blackfan
    Intervention:   Drug: Eltrombopag
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    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 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
    Intervention:  
    Sponsors:   St. Jude Children’s Research Hospital;   Boston Children’s Hospital;   University of Memphis
    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
  • Multi-Center Study of Iron Overload: Survey Study (MCSIO)
    Conditions:   Sickle Cell Disease;   Thalassemia;   Diamond-Blackfan Anemia
    Intervention:  
    Sponsors:   UCSF Benioff Children’s Hospital Oakland;   University College London (UCL) Cancer Institute;   Universitätsklinikum Hamburg-Eppendorf;   Medical University Innsbruck;   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
    Completed
  • Multi-Center Study of Iron Overload: Pilot Study
    Conditions:   Sickle Cell Disease;   Thalassemia;   Diamond-Blackfan Anemia
    Intervention:  
    Sponsors:   UCSF Benioff Children’s Hospital Oakland;   University College London (UCL) Cancer Institute;   Universitätsklinikum Hamburg-Eppendorf;   Medical University Innsbruck;   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
    Completed
  • 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 hematopoieticprogenitor 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