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Wilson's Disease clinical trials at UCLA

2 in progress, 0 open to eligible people

Showing trials for
  • Phase 1/2/3 Study of UX701 Gene Therapy in Adults With 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 5368361, California 5332921 and other locations

  • Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

    Sorry, not yet accepting patients

    The aim of this study is to inform and improve future clinical trials in Wilson Disease (WD) by better understanding how patients with WD are living with and managing the disease, and by identifying key factors that shape their decisions to participate in clinical research.

    Los Angeles 5368361, California 5332921 and other locations

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