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Wilson's Disease clinical trials at University of California Health

3 in progress, 2 open to eligible people

Showing trials for
  • VTX-801 in Adult Patients With Wilson's Disease

    “Become involved in the GATEWAY Clinical Trial (Gene Therapy for Wilson's Disease)”

    open to eligible people ages 18-65

    The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.

    at UC Davis

  • Real World Evidence Study in Subjects With Wilson's Disease

    open to eligible people ages 18 years and up

    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.

    at UC Davis

  • 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.

    at UC Davis UCLA

Our lead scientists for Wilson's Disease research studies include .

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