RAAV2/5-hNAGLU

( 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	2

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	NCT03300453 (ClinicalTrials.gov)	September 17, 2013	15/6/2016	Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome	Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-ass ...	Sanfilippo Syndrome B	Drug: rAAV2/5-hNAGLU	UniQure Biopharma B.V.	Venn Life Sciences;Institut Pasteur	Completed	18 Months	60 Months	All	4	Phase 1/Phase 2	France
2	EUCTR2012-000856-33-FR (EUCTR)		15/05/2014	Intracerebral Gene therapy in children with Sanfilippo type B syndrome	A phase I/II, open-label, study of intracerebral administration of adeno-associated viral vector con ...	Mucopolysaccharidosis III B MedDRA version: 17.0;Level: PT;Classification code 10056890;Term: Mucopo ...	Product Name: rAAV2/5-hNaGlu	Institut Pasteur	NULL	NA			Female: yes Male: yes		Phase 1;Phase 2	France

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