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.

  • 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 Leukaemia;   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 – verified February 2017
  • 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
    Recruiting – verified September 2016
  • Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
    Conditions:   Stem Cell Transplantation;   Bone Marrow Transplantation;   Peripheral Blood Stem Cell Transplantation;   Allogeneic Transplantation;   Genetic Diseases;   Thalassemia;   Pediatrics;   Diamond-Blackfan Anemia;   Combined Immune Deficiency;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   X-linked Lymphoproliferative Disease;   Metabolic Diseases
    Interventions:   Drug: Cyclophosphamide Dose Level 1;   Drug: Cyclophosphamide Dose Level 2;   Drug: Cyclophosphamide Dose Level 3;   Drug: Cyclophosphamide Dose Level 4
    Sponsors:   Children’s Hospital Los Angeles;   Lucile Packard Children’s Hospital
    Terminated – verified January 2017
  • 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 – verified February 2017
  • Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT
    Conditions:   Primary Immunodeficiency Syndromes;   Congenital Bone Marrow Failure Syndromes;   Inherited Metabolic Disorders (IMD);   Hereditary Anemias;   Patients With Sickle Disease Presenting Specific Symptoms
    Interventions:   Drug: Hydroxyurea;   Drug: Alemtuzumab;   Drug: Fludarabine;   Drug: Melphalan;   Drug: Thiotepa
    Sponsor:   University of Pittsburgh
    Recruiting – verified March 2017
  • Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
    Conditions:   Chronic Kidney Disease;   Acute Myeloid Leukemia (AML);   Acute Lymphoblastic Leukemia (ALL);   Chronic Myelogenous Leukemia (CML);   Chronic Lymphocytic Leukemia (CLL);   Non-Hodgkin’s Lymphoma (NHL);   Hodgkin Disease;   Multiple Myeloma;   Myelodysplastic Syndrome (MDS);   Aplastic Anemia;   AL Amyloidosis;   Diamond Blackfan Anemia;   Myelofibrosis;   Myeloproliferative Disease;   Sickle Cell Anemia;   Autoimmune Diseases;   Thalassemia
    Intervention:   Procedure: Haploidentical Bone Marrow/Kidney
    Sponsor:   Massachusetts General Hospital
    Recruiting – verified March 2017
  • Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
    Conditions:   Accelerated Phase Chronic Myelogenous Leukemia;   Adult Acute Lymphoblastic Leukemia in Remission;   Adult Acute Myeloid Leukemia in Remission;   Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;   Adult Acute Myeloid Leukemia With Del(5q);   Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);   Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);   Adult Acute Myeloid Leukemia With t(16;16)(p13;q22);   Adult Acute Myeloid Leukemia With t(8;21)(q22;q22);   Adult Grade III Lymphomatoid Granulomatosis;   Adult Nasal Type Extranodal NK/T-cell Lymphoma;   Anaplastic Large Cell Lymphoma;   Angioimmunoblastic T-cell Lymphoma;   Aplastic Anemia;   Burkitt Lymphoma;   Childhood Acute Lymphoblastic Leukemia in Remission;   Childhood Acute Myeloid Leukemia in Remission;   Childhood Chronic Myelogenous Leukemia;   Childhood Diffuse Large Cell Lymphoma;   Childhood Grade III Lymphomatoid Granulomatosis;   Childhood Immunoblastic Large Cell Lymphoma;   Childhood Myelodysplastic Syndromes;   Childhood Nasal Type Extranodal NK/T-cell Lymphoma;   Chronic Myelomonocytic Leukemia;   Chronic Phase Chronic Myelogenous Leukemia;   Congenital Amegakaryocytic Thrombocytopenia;   Diamond-Blackfan Anemia;   Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;   Hepatosplenic T-cell Lymphoma;   Juvenile Myelomonocytic Leukemia;   Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;   Nodal Marginal Zone B-cell Lymphoma;   Paroxysmal Nocturnal Hemoglobinuria;   Peripheral T-cell Lymphoma;   Polycythemia Vera;   Post-transplant Lymphoproliferative Disorder;   Previously Treated Myelodysplastic Syndromes;   Primary Myelofibrosis;   Recurrent Adult Acute Lymphoblastic Leukemia;   Recurrent Adult Acute Myeloid Leukemia;   Recurrent Adult Burkitt Lymphoma;   Recurrent Adult Diffuse Large Cell Lymphoma;   Recurrent Adult Diffuse Mixed Cell Lymphoma;   Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;   Recurrent Adult Grade III Lymphomatoid Granulomatosis;   Recurrent Adult Hodgkin Lymphoma;   Recurrent Adult Immunoblastic Large Cell Lymphoma;   Recurrent Adult Lymphoblastic Lymphoma;   Recurrent Adult T-cell Leukemia/Lymphoma;   Recurrent Childhood Acute Lymphoblastic Leukemia;   Recurrent Childhood Acute Myeloid Leukemia;   Recurrent Childhood Anaplastic Large Cell Lymphoma;   Recurrent Childhood Grade III Lymphomatoid Granulomatosis;   Recurrent Childhood Large Cell Lymphoma;   Recurrent Childhood Lymphoblastic Lymphoma;   Recurrent Childhood Small Noncleaved Cell Lymphoma;   Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;   Recurrent Grade 1 Follicular Lymphoma;   Recurrent Grade 2 Follicular Lymphoma;   Recurrent Grade 3 Follicular Lymphoma;   Recurrent Mantle Cell Lymphoma;   Recurrent Marginal Zone Lymphoma;   Recurrent Mycosis Fungoides/Sezary Syndrome;   Recurrent Small Lymphocytic Lymphoma;   Recurrent/Refractory Childhood Hodgkin Lymphoma;   Refractory Chronic Lymphocytic Leukemia;   Refractory Hairy Cell Leukemia;   Refractory Multiple Myeloma;   Secondary Acute Myeloid Leukemia;   Secondary Myelodysplastic Syndromes;   Secondary Myelofibrosis;   Severe Combined Immunodeficiency;   Severe Congenital Neutropenia;   Shwachman-Diamond Syndrome;   Splenic Marginal Zone Lymphoma;   T-cell Large Granular Lymphocyte Leukemia;   Waldenstrom Macroglobulinemia;   Wiskott-Aldrich Syndrome
    Interventions:   Drug: fludarabine phosphate;   Drug: melphalan;   Radiation: total-body irradiation;   Drug: tacrolimus;   Drug: mycophenolate mofetil;   Drug: methotrexate;   Other: laboratory biomarker analysis;   Procedure: allogeneic hematopoietic stem cell transplantation;   Procedure: peripheral blood stem cell transplantation
    Sponsor:   Roswell Park Cancer Institute
    Active, not recruiting – verified March 2017
  • Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
    Conditions:   Anemia, Sickle Cell;   Complex and Transfusion-dependent Hemoglobinopathies;   Thalassemia;   Diamond-Blackfan Anemia;   Bone Marrow Failure Syndromes Characterized by Severe Chronic Anemia;   Alpha-Thalassemia;   Beta-Thalassemia
    Intervention:   Biological: Enriched Hematopoetic Stem Cell Infusion
    Sponsors:   University of Louisville;   Duke University
    Suspended – verified March 2017
  • A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
    Condition:   Transfusional Iron Overload
    Intervention:   Drug: ICL670
    Sponsor:   Novartis Pharmaceuticals
    Completed – verified February 2017
  • Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
    Condition:   Non-Malignant
    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 – verified February 2017
  • Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
    Conditions:   Hemoglobinopathies;   Myelodysplastic Syndromes;   Other Inherited or Acquired Anaemia;   MPD Syndrome;   Diamond-Blackfan Anemia;   Other Rare Anaemias;   Transfusional Iron Overload
    Intervention:   Drug: deferasirox
    Sponsor:   Novartis
    Completed – verified February 2017
  • Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
    Conditions:   Congenital Hemolytic Anemia;   Diamond-Blackfan Anemia
    Interventions:   Procedure: Radiotherapy;   Drug: Alemtuzumab (Campath );   Drug: Sirolimus (Rapamune )
    Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
    Recruiting – verified January 3, 2017
  • Cancer in Inherited Bone Marrow Failure Syndromes
    Conditions:   Aplastic Anemia;   Head and Neck Neoplasms;   Bone Marrow Failure
    Intervention:  
    Sponsor:   National Cancer Institute (NCI)
    Recruiting – verified February 8, 2017