Wilson's Disease clinical trials at University of California Health
3 research studies open to eligible people
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
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 UC Davis UCLA
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 UCLA
Our lead scientists for Wilson's Disease research studies include Valentina Medici, Dr Sammy Saab Jeff Bronstein.
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