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Wilson's Disease clinical trials at UCLA

2 in progress, 1 open to eligible people

Showing trials for
  • 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 .

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