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.

  • Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
    Conditions:   Acute Lymphoblastic Leukemia;   Leukemia, Acute Myeloid (AML), Child;   Lymphoma, Non-Hodgkin;   Myelodysplastic Syndromes;   Primary Immunodeficiency;   Anemia, Aplastic;   Hemoglobinopathies;   Cytopenia;   Fanconi Anemia;   Diamond Blackfan Anemia;   Thalassemia;   Anemia, Sickle Cell
    Interventions:   Drug: Rimiducid;   Biological: rivogenlecleucel
    Sponsor:   Bellicum Pharmaceuticals
    Enrolling by invitation
  • T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease and Thalassemia
    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:   Beth Carella, DO
    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
    Sponsor:   University of Florida
    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;   Baylor College of Medicine;   University of Memphis
    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
    Sponsor:   Bellicum Pharmaceuticals
    Terminated
  • 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
  • Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant
    Conditions:   Acute Lymphoblastic Leukemia;   Leukemia, Acute Myeloid (AML), Child;   Lymphoma, Non-Hodgkin;   Myelodysplastic Syndrome;   Primary Immunodeficiency;   Anemia, Aplastic;   Osteopetrosis;   Hemoglobinopathies;   Cytopenia;   Fanconi Anemia;   Diamond Blackfan Anemia;   Thalassemia;   Anemia, Sickle Cell
    Interventions:   Biological: BPX-501 T cells;   Drug: rimiducid
    Sponsor:   Bellicum Pharmaceuticals
    Active, not 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
    Sponsor:   Emory University
    Active, not 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
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    Completed
  • 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
    Sponsors:   Masonic Cancer Center, University of Minnesota;   National Marrow Donor Program
    Active, not recruiting
  • Diamond Blackfan Anemia Registry (DBAR)
    Conditions:   Anemia;   Blood Disease
    Intervention:  
    Sponsors:   Northwell Health;   National Heart, Lung, and Blood Institute (NHLBI)
    Recruiting
  • Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
    Conditions:   Congenital Hemolytic Anemia;   Diamond-Blackfan Anemia
    Interventions:   Procedure: Peripheral blood hematopoietic progenitor cell (PBPC) transplant;   Drug: Alemtuzumab + Sirolimus + Peripheral blood hematopoietic progenitor cell (PBPC) transplant
    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