A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Active, not recruiting · Phase 1, Phase 2 · NCT04884815
Conditions: Wilson Disease
At a glance
- Research payments reported, 2025
- $495,859
- U.S. investigators named
- 12
- Sites paid
- 13
- Planned or actual enrollment
- 82
About the trial
- Sponsor
- Ultragenyx Pharmaceutical Inc
- Tested
- UX701 (genetic), Standard of Care (SOC) (drug)
- Dates
- Started 2021-09-27; completion 2034-03
- Results posted
- Not yet
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.
From ClinicalTrials.gov, last updated 2026-09-25. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ultragenyx Pharmaceutical Inc. reported $495,859 in research payments for this study in 2025 (112 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Jeff M. Bronstein, MD Neurology · Los Angeles, CA
- Paul Y. Kwo, M.D. Hepatology · Stanford, CA
- Valentina Medici, M.D. Gastroenterology · Sacramento, CA
- Cynthia Levy, MD Hepatology · Miami, FL
- Danny Bega, M.D. Neurology · Chicago, IL
- Craig S. Lammert, MD Gastroenterology · Indianapolis, IN
- Russell Goodman, M.D. Gastroenterology · Boston, MA
- Frederick K. Askari, MD, PHD Gastroenterology · Ann Arbor, MI
- Andrew J. Muir, M.D. Gastroenterology · Durham, NC
- Andrew E. Scanga, MD Hepatology · Nashville, TN
- Juan F. Gallegos-orozco, M.D. Hepatology · Salt Lake City, UT
- Sihoun Hahn, MD, PHD Medical Genetics · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of California Davis Medical Center, CA $128,712
- University of Michigan Health System, MI $104,429
- Massachusetts General Hospital, MA $57,812
- Ronald Reagan UCLA Medical Center, CA $40,177
- Stanford University $39,823
- Indiana University $34,065
- Northwestern University $29,414
- University of Utah Hospital and Clinics, UT $15,850
- Duke University Medical Center $15,761
- Seattle Children's Hospital, WA $13,903
- Regents of the University California Los Angeles $8,412
- Vanderbilt University Medical Center, TN $4,900
- University of Miami Miller School $2,601
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.