Ultragenyx UX701-CL301 (Wilson Disease)

Purpose of this Study

We are doing this study to find out if an experimental drug called UX701 (the study drug) is a safe and effective option for people with Wilson disease. We also want to know if an experimental laboratory test called AAV9 DetectCDx can help us identify patients who might benefit by taking the study drug.

Who Can Participate?

Eligibility

Adults ages 18+ who:
  • Are diagnosed with Wilson Disease
  • Are being treated with a copper chelator and/or zinc therapy
  • Have never received a liver transplant and do not have current plans for a transplant
  • Do not have a history of any of the following: ascites (fluid buildup in the abdomen), esophageal bleeding, hepatic encephalopathy, chronic kidney disease, or HIV
For more information, contact the study team at christy.walters@duke.edu.

Age Range

18-110

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

If you choose to join this study, you will begin during Stage 1 or Stage 2. In either of these stages, you will get a random assignment (by chance) to either get the study drug or a placebo (inactive substance with no drug in it). Whether or not you take part in Stage 1 or Stage 2 will depend on when you join the study. After you finish Stage 1 or Stage 2, you will have the option to take part in Stage 3. Participants in Stage 2 can proceed to Stage 3 immediately, but participants in Stage 1 might have to wait before proceeding to Stage 3. Everyone who participates in Stage 3 will get the study drug.

Locations

Duke University Hospital
Pickett Road Research

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

No

Study Details

Full Title

A Randomized, Double-Blind, Placebo-Controlled, Multicenter,
Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease

Principal Investigator

Andrew
Muir

Protocol Number

PRO00108062

NCT ID

NCT04884815

Phase

I/II

Enrollment Status

Open to Enrollment
ClinicalTrials.gov