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
  • 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
  • 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
  • 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
  • CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
    Conditions:   Chronic Myeloid Leukemia (CML);   Acute Myelogenous Leukemia (AML);   Myelodysplastic Syndrome (MDS);   Juvenile Myelomonocytic Leukemia (JMML);   Acute Lymphoblastic Leukemia (ALL);   Lymphoma (Hodgkin’s and Non-Hodgkin’s)
    Interventions:   Device: CliniMACS CD34+ Reagent System;   Drug: Thiotepa;   Drug: Cyclophosphamide;   Drug: Alemtuzumab;   Drug: Tacrolimus;   Drug: Melphalan;   Drug: Busulfan;   Drug: Fludarabine;   Drug: Methylprednisolone
    Sponsor:   Diane George
    Recruiting
  • CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
    Conditions:   Bone Marrow Failure Syndrome;   Severe Aplastic Anemia;   Severe Congenital Neutropenia;   Amegakaryocytic Thrombocytopenia;   Diamond-Blackfan Anemia;   Schwachman Diamond Syndrome;   Primary Immunodeficiency Syndromes;   Acquired Immunodeficiency Syndromes;   Histiocytic Syndrome;   Familial Hemophagocytic Lymphocytosis;   Lymphohistiocytosis;   Macrophage Activation Syndrome;   Langerhans Cell Histiocytosis (LCH);   Hemoglobinopathies;   Sickle Cell Disease;   Sickle Cell-beta-thalassemia
    Intervention:   Biological: CD34 Stem Cell Selection Therapy
    Sponsor:   Diane George
    Active, not 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
  • 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
  • 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