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 : 37 薬物数 : 110 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 124

  
2 trials found
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1NCT03733249
(ClinicalTrials.gov)
January 20172/11/2018Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyFollow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute 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 CellDrug: Rimiducid;Biological: rivogenlecleucelBellicum PharmaceuticalsNULLEnrolling by invitation1 Month18 YearsAll193Phase 1/Phase 2Italy;Saudi Arabia;United Kingdom
2NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute 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 CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNULLActive, not recruiting1 Month18 YearsAll193Phase 2Italy;United Kingdom;Germany;Spain;United States