Wilson's Disease clinical trials at UCLA
2 in progress, 1 open to eligible people
UX701 Gene Transfer for the Treatment of Wilson Disease
open to eligible people ages 18 years and up
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
Real World Evidence Study in Subjects With Wilson's Disease
Sorry, not currently recruiting here
This non-interventional Real-World Evidence (RWE) study aims to describe non-ceruloplasmin copper values obtained using a new NCC Speciation assay by taking a small (up to 10mLs) volume of additional blood from patients with Wilson's Disease, around the time when routine blood sampling is expected to be scheduled by the treating physician. Data will be collected over an approximate 12-month period.
Los Angeles, California and other locations
Our lead scientists for Wilson's Disease research studies include Jeff Bronstein Sammy Saab.
Last updated: