Wilson's Disease clinical trials at UC Davis
3 research studies open to eligible people
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.
Sacramento, California and other locations
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
open to eligible people ages 18 years and up
The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at least 1 year with standard of care (that is, trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840 will be assessed at Week 48.
Sacramento, California and other locations
ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
open to eligible people ages 12 years and up
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.
Los Angeles, California and other locations
Our lead scientists for Wilson's Disease research studies include Valentina Medici, Dr.
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