A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Public ClinicalTrials.gov record NCT04884815. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04884815
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 82 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 27, 2021 (Actual)
- Primary completion
- Mar 2029 (Estimated)
- Completion
- Mar 2034 (Estimated)
- Last update posted
- Sep 25, 2026
2021 – 2034
United States locations
- U.S. sites
- 12
- U.S. states
- 10
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | 90095 | — |
| Stanford University | Redwood City | California | 94063 | — |
| University of California Davis | Sacramento | California | 95817-1348 | — |
| Northwestern University | Chicago | Illinois | 60611 | — |
| Indiana University | Indianapolis | Indiana | 46202 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| University of Michigan | Ann Arbor | Michigan | 48109 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37212-2700 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04884815, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 25, 2026 · Synced Oct 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04884815 live on ClinicalTrials.gov.