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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
  • A Phase I/II Study of 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

  • Study of 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.

    at UCLA

  • Effectiveness and Safety of Experimental ALXN1840 Versus Standard of Care for Wilson Disease (copper buildup)

    Sorry, in progress, not accepting new patients

    The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation Period. In addition, efficacy and safety will be evaluated during an optional 60-month Extension Period.

    at UCLA

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

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