Intracerebral administration of AAVrh.10cuARSA

( DrugBank: - / KEGG DRUG: - )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	1

19. ライソゾーム病

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

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01801709 (ClinicalTrials.gov)	June 2014	28/1/2013	Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy	A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication ...	Metachromatic Leukodystrophy	Genetic: intracerebral administration of AAVrh.10cuARSA	Institut National de la Santé Et de la Recherche Médicale, France	European Leukodystrophy Association;Assistance Publique - Hôpitaux de Paris	Active, not recruiting	6 Months	5 Years	All	5	Phase 1/Phase 2	France

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