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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1NCT01801709
(ClinicalTrials.gov)
June 201428/1/2013Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic LeukodystrophyA Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.Metachromatic LeukodystrophyGenetic: intracerebral administration of AAVrh.10cuARSAInstitut National de la Santé Et de la Recherche Médicale, FranceEuropean Leukodystrophy Association;Assistance Publique - Hôpitaux de ParisActive, not recruiting6 Months5 YearsAll5Phase 1/Phase 2France