drugset / Trial / NCT04884815
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
RandomizedSequentialSingle-blindTreatment
Summary
The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation.
Timeline
- Start
- 2021-09-27
- Primary completion
- 2029-03
- Completion
- 2034-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RIVUNATPAGENE MIZIPARVOVEC | Gene therapy (AAV / viral vector) | — | Intravenous |