Wilson's Disease clinical trials at UCLA

1 in progress, 0 open to eligible people

UX701 Gene Transfer for the Treatment of Wilson Disease
Sorry, in progress, not accepting new patients
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.
Los Angeles, California and other locations

Last updated: November 25, 2024