326. 大理石骨病 Osteopetrosis Clinical trials / Disease details


臨床試験数 : 18 薬物数 : 43 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 82

  
2 trials found
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1NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell TransplantPhase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRaß+ T Cells in Paediatric Patients Affected by Haematological 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 YearsAll187Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
2EUCTR2014-000584-41-GB
(EUCTR)
15/07/2015Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantationPhase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT Hematological disorders (ALL; AML; Non-Hodgkin lymphoma; Myelodysplastic syndromes; Congenital immune deficiencies; Severe aplastic anemia; Fanconi anemia; Osteopetrosis; Selected cases of hemoglobinopathies)
MedDRA version: 20.0;Level: HLGT;Classification code 10018849;Term: Haematological disorders NEC;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: BPX-501 cells
INN or Proposed INN: Rivogenlecleucel
Other descriptive name: BPX-501
Product Name: rimiducid
INN or Proposed INN: Rimiducid
Other descriptive name: AP1903 A594
Bellicum Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
175Phase 2Spain;Italy;United Kingdom