Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease
Status:
Recruiting
Trial end date:
2031-01-01
Target enrollment:
Participant gender:
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.