Drugs / AAVRh.10CUhCLN2
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT01161576 | Aug 2010 → Jan 2016 | late infantile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder | Weill Medical College of Cornell University | Completed | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AAVRh.10CUhCLN2 | “administer to the brain an experimental drug called AAVRh.10CUhCLN2, a gene transfer vector” NCT01161576 ↗ |
| Action | Restore | “the aim of this study is to add a normal copy of the CLN2 gene to the brain of affected children to try to reverse death of cells in the brain” NCT01161576 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Direct CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA” NCT01161576 ↗ |
| Route | Other | “Children with Batten disease will get the drug injected into the brain” NCT01161576 ↗ |
| Target | CLN2 | “the aim of this study is to add a normal copy of the CLN2 gene to the brain of affected children” NCT01161576 ↗ |