Drugs / AVALOTCAGENE ONTAPARVOVEC
Trials 2 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02991144 | Jul 2017 → Dec 2021 | ornithine carbamoyltransferase deficiency | Ultragenyx Pharmaceutical Inc | Completed | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT05345171 | Oct 2022 → Sep 2027 expected | ornithine carbamoyltransferase deficiency | Ultragenyx Pharmaceutical Inc | Active not recruiting | No outcome recorded |
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AVALOTCAGENE ONTAPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650252 ↗ |
| Known as | DTX301 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05345171 ↗ |
| Known as | KFB12DX3PF | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650252 ↗ |
| Known as | scAAV8OTC | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02991144 ↗ |
| Action | Restore | “improvement of ornithine transcarbamylase (OTC) function” NCT05345171 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC)” NCT02991144 ↗ |
| Route | Intravenous | “single peripheral IV infusion” NCT02991144 ↗ |
| Target | OTC | “non-replicating, recombinant scAAV8 encoding human ornithine transcarbamylase (OTC)” NCT02991144 ↗ |