AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 19 | Lysosomal storage disease | 1 |
19. Lysosomal storage disease
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 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 Lipofuscinosis | Biological: AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies;Biological: AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies | Weill Medical College of Cornell University | National Institutes of Health (NIH) | Completed | 2 Years | 18 Years | All | 12 | Phase 1 | United States |