AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease1

19. Lysosomal storage disease


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01161576
(ClinicalTrials.gov)
August 19, 201022/3/2010Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)Batten Disease;Late-Infantile Neuronal Ceroid LipofuscinosisBiological: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;Biological: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copiesWeill Medical College of Cornell UniversityNational Institutes of Health (NIH)Completed2 Years18 YearsAll12Phase 1United States