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
  • AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
    Conditions:   Acute Leukemia;   Severe Aplastic Anemia;   Non-hodgkin Lymphoma;   Hodgkin Lymphoma;   Kostmann;   Diamond Blackfan Anemia;   Amegakaryocytic Thrombocytopenia;   Sickle Cell Disease;   Beta-Thalassemia
    Intervention:   Drug: alpha beta depletion
    Sponsor:   Mitchell Cairo
    Not yet recruiting
  • The Use of Trifluoperazine in Transfusion Dependent DBA
    Conditions:   Diamond Blackfan Anemia;   Pure Red Cell Aplasia
    Intervention:   Drug: Trifluoperazine
    Sponsor:   Adrianna Vlachos
    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:   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
    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
    Sponsors:   Roswell Park Cancer Institute;   National Cancer Institute (NCI)
    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
  • 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
    Active, not 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
  • 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
    Sponsor:   New York Medical College
    Active, not recruiting
  • Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
    Condition:   Diamond Blackfan Anemia
    Interventions:   Drug: Sotatercept;   Drug: Sotatercept with prednisone boost
    Sponsor:   Northwell Health
    Recruiting
  • Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
    Conditions:   Diamond Blackfan Anemia;   Blackfan Diamond Syndrome;   DBA;   Congenital Hypoplastic Anemia;   Pure Red Cell Aplasia
    Intervention:   Drug: leucine
    Sponsor:   Northwell Health
    Active, not recruiting
  • Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
    Condition:   Non-Neoplastic Hematologic and Lymphocytic Disorder
    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
    Sponsors:   Fred Hutchinson Cancer Research Center;   medac GmbH;   National Cancer Institute (NCI);   National Heart, Lung, and Blood Institute (NHLBI)
    Recruiting
  • A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
    Condition:   Transfusion-dependent Iron Overload
    Intervention:   Drug: Deferasirox
    Sponsor:   Novartis Pharmaceuticals
    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
    Completed
  • 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;   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