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Gene therapy could free wilson disease patients from daily pills

NCT ID NCT04884815

Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy called UX701 for adults with Wilson disease, a genetic condition that causes copper buildup. The goal is to see if it is safe and can help the body control copper levels, possibly allowing patients to take fewer or no daily medications. About 82 participants will receive either the gene therapy or standard care.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
UX701 (rivunatpagene miziparvovec) gene therapy
What this could lead to
If successful, this gene therapy could allow people with Wilson disease to reduce or stop taking daily copper-control medications, simplifying their treatment.
What could go wrong
This is an early-phase trial (Phase 1/2) with only 82 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues, and it may not work as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 82 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2021

Expected to finish

Mar 2034

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening, with no medication or dose changes for at least 2 months at screening. * Ongoing restriction of high copper containing foods for at least 2 months at Screening and continued through study participation. * Willing and able to comply with all study procedures and requirements, including frequent blood collection, total urine collection over a 24-hour period, patient-reported outcome assessments, and long-term follow-up Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV9 capsid. * Stage 1 only: History of copper chelator or zinc therapy noncompliance, in the Investigator's judgment, within 6 months prior to Screening. * History of liver transplant. * Active decompensated hepatic cirrhosis or history of hepatic encephalopathy. * Significant hepatic inflammation as evidenced by laboratory abnormalities. * Model for End-Stage Liver Disease (MELD) score \> 13. * Hemoglobin \< 9 g/dL * Presence of Stage 3 or higher chronic kidney disease based on estimated glomerular filtration rate \< 60 mL/min/1.73 m2. * Marked neurological deficit or compromise that, in the Investigator's opinion, would interfere with the subject's safety or ability to participate in the study. * Moderate to severe depression, recent or active suicidal ideation with intent or suicidal behavior, psychosis, or unstable psychiatric illness. * Known hypersensitivity to UX701 or its excipients, copper chelators, zinc, rituximab, tacrolimus, corticosteroids, or eculizumab that, in the Investigator's judgement, places the participant at increased risk for adverse events. * Participation in another gene transfer study or use of another gene transfer product before or during study participation. * Subjects with known hypersensitivity to amide-containing local anesthetics are excluded from participating in the optional liver biopsy substudy. Note: Other protocol defined Inclusion/ Exclusion criteria may apply

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Conditions

The condition(s) this trial relates to.

Wilson disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Wilson disease

Contacts and locations

Locations

  • Centro Hospitalar Universitário Lisboa Norte

    Lisbon, Lisbon District, 1649-035, Portugal

  • Centro Hospitalar Universitário de São João

    Porto, Porto District, 4200-319, Portugal

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Hospital Universitario Vall d'Hebron - PPDS

    Barcelona, 08035, Spain

  • Indiana University

    Indianapolis, Indiana, 46202, United States

  • Kings College NHS Foundation

    London, Surrey, SE5 9RS, United Kingdom

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Stanford University

    Redwood City, California, 94063, United States

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University of California Davis

    Sacramento, California, 95817-1348, United States

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37212-2700, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.