284. ダイアモンド・ブラックファン貧血 Diamond-Blackfan anemia Clinical trials / Disease details


臨床試験数 : 36 薬物数 : 95 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

  
6 trials found
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1NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone 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;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchActive, not recruiting3 Months39 YearsAll6Phase 1/Phase 2United States
2NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A21 YearsAll35Phase 2United States
3NCT00176852
(ClinicalTrials.gov)
June 200212/9/2005Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramCompletedN/A50 YearsAll22Phase 2/Phase 3United States
4NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
5NCT00176878
(ClinicalTrials.gov)
June 200012/9/2005Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLCompletedN/A35 YearsAll10Phase 2/Phase 3United States
6NCT00290628
(ClinicalTrials.gov)
October 19999/2/2006Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic CancerTransplantation of Umbilical Cord Blood From Related and Unrelated DonorsChronic Myeloproliferative Disorders;Diamond-blackfan Anemia;Fanconi Anemia;Graft Versus Host Disease;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative DiseasesDrug: cyclophosphamide;Drug: cyclosporine;Drug: filgrastim;Drug: melphalan;Drug: methylprednisolone;Drug: mycophenolate mofetil;Procedure: radiation therapy;Procedure: umbilical cord blood transplantation;Drug: anti-thymocyte globulin;Drug: busulfanMasonic Cancer Center, University of MinnesotaNational Cancer Institute (NCI)TerminatedN/A45 YearsAll43N/AUnited States