19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 854 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 69 - 標的パスウェイ数 : 190

  
1 trial found
No.TrialIDDate_
enrollment
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agemin
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agemax
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PhaseCountries
1NCT03639844
(ClinicalTrials.gov)
November 6, 201717/8/2018BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 StudyExpanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR aß+T Cells In Pediatric Patients Affected by Hematological and Other DisordersHurler Syndrome;Inherited Metabolic Disorder;Lysosomal Storage Disorder;Metachromatic Leukodystrophy;Inborn Errors of MetabolismBiological: rivogenlecleucel;Drug: rimiducidBellicum PharmaceuticalsNULLNo longer available3 Months21 YearsAllUnited States