AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies

( DrugBank: - / KEGG DRUG: - )

1 disease

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	1

19. Lysosomal storage disease

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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	NCT01161576 (ClinicalTrials.gov)	August 19, 2010	22/3/2010	Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipo ...	Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Ser ...	Batten Disease;Late-Infantile Neuronal Ceroid Lipofuscinosis	Biological: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;Biological: AAVrh.10CUhCLN2 vector 2.85x1 ...	Weill Medical College of Cornell University	National Institutes of Health (NIH)	Completed	2 Years	18 Years	All	12	Phase 1	United States

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