Drugs / GNT0003
GNT0003
Trials 2 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT06518005 | Nov 2024 → Sep 2026 expected | Crigler-Najjar syndrome | Genethon | Recruiting | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT03466463 | Mar 2018 → Mar 2026 overdue | Crigler-Najjar syndrome | Genethon | Recruiting | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | GNT0003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06518005 ↗ |
| Action | Restore | “aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene” NCT06518005 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “an adeno-associated virus serotype 8 vector encoding UGT1A1” PMID 37585628 ↗ Aug 2023 |
| Route | Intravenous | “a single intravenous infusion” PMID 37585628 ↗ Aug 2023 |
| Target | UGT1A1 | “an adeno-associated virus serotype 8 vector encoding UGT1A1” PMID 37585628 ↗ Aug 2023 |