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


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
1 trial found
No.TrialIDDate_
enrollment
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registration
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agemin
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agemax
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PhaseCountries
1NCT01560182
(ClinicalTrials.gov)
April 9, 201016/3/2012Gene Therapy for Metachromatic Leukodystrophy (MLD)A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic LeukodystrophyLysosomal Storage Disease;Metachromatic LeukodystrophyGenetic: OTL-200 Gene TherapyOrchard TherapeuticsOspedale San RaffaeleActive, not recruitingN/A7 YearsAll20Phase 1/Phase 2Italy