Drugs / rAAV1-CMV-GAA
rAAV1-CMV-GAA
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT00976352 | Sep 2010 → Dec 2015 | glycogen storage disease II | University of Florida | Completed | No outcome recorded |
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAV1-CMV-GAA | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00976352 ↗ |
| Known as | rAAV1-CMV-hGAA | “a modified virus, adeno-associated virus (AAV), has been engineered to carry a normal copy of the GAA gene, known as rAAV1-CMV-hGAA, which is used to place normal copies of the...” NCT00976352 ↗ |
| Known as | rAAV1-hGAA | “Here we present 180-day safety and ventilatory outcomes for five ventilator-dependent children in a phase I/II clinical trial of AAV-mediated GAA gene therapy (rAAV1-hGAA)...” PMID 23570273 ↗ Jun 2013 |
| Action | Restore | “which is used to place normal copies of the GAA gene into diaphragm muscle cells” NCT00976352 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “a modified virus, adeno-associated virus (AAV), has been engineered to carry a normal copy of the GAA gene” NCT00976352 ↗ |
| Route | Intramuscular | “rAAV1-CMV-GAA via intramuscular injection into the diaphragm” NCT00976352 ↗ |