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
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1NCT00151216
(ClinicalTrials.gov)
June 20046/9/2005Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid LipofuscinosisAdministration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children With Late Infantile Neuronal Ceroid LipofuscinosisBatten Disease;Late Infantile Neuronal Ceroid LipofuscinosisBiological: AAV2CUhCLN2 (3x10^12 particle units)Weill Medical College of Cornell UniversityNathan's Battle FoundationCompleted3 Years18 YearsAll10Phase 1United States