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One-Time gene shot aims to tame rare copper disorder

NCT ID NCT04537377

Disease control Sponsor: Vivet Therapeutics SAS 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 early-stage trial tests a single dose of VTX-801 gene therapy in 4 adults with Wilson's disease, a rare condition where copper builds up in the body. The goal is to see if the treatment is safe and can help control copper levels, possibly allowing patients to stop their usual medications. Researchers will follow participants for up to 5 years.

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

4 people

The number who actually took part.

Started

Sep 2021

Expected to finish

Jun 2029

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 to 65 years

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.

Main Inclusion Criteria: * Male or female aged 18 and 65 years inclusive * Confirmed diagnosis of WD * Treated for WD according to international recommendations with no current evidence for inadequate treatment * Stable WD for ≥ 1 year, defined as: (i) No significant change in neurologic examination and in status of mood disorder and (ii) Stable laboratory parameters used to assess copper metabolism Main Exclusion Criteria: * ALT level ≥ 2 ULN that is not readily explained by extrinsic factors * Total bilirubin \> 1.5 x ULN in the absence of proven Gilbert's syndrome; in case of Gilbert's syndrome, direct bilirubin \> ULN * INR \> 1.2 * Any signs of liver cirrhosis decompensation, including gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrollment visit * Patient has moderate or severe renal impairment defined as eGFR CKD-EPI \< 60 mL/min/1.73 m2, or patient has nephritis or nephrotic syndrome * Any history or current evidence of HIV-1, HIV-2, HTLV 1, or HTLV-2 infection * Any history or current evidence of hepatitis B infection * Any history of hepatitis C infection, unless previous viral RNA assays in two samples, collected at least 6 months apart, are negative * Positive QuantiFERON®-TB Gold tuberculosis test result * Any concomitant disorder/condition - including hepatic disorders - or treatment possibly interfering with the conduct or evaluation of the study * Any history of diabetes * Pregnancy or breastfeeding * Body Mass Index ≥ 35 kg/m2 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's disease

Contacts and locations

Locations

  • Aarhus University Hospital

    Aarhus, 8200, Denmark

  • Advent Health

    Orlando, Florida, 32803, United States

  • Royal Surrey County Hospital

    Guildford, Surrey, GU2 7XX, United Kingdom

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • University Hospital Essen

    Essen, 45147, Germany

  • University of Michigan Health System

    Ann Arbor, Michigan, 48109, United States

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75235, United States

  • Universitätsklinikum Tübingen (UKT)

    Tübingen, 72076, Germany

  • Wake Forest School of Medicine

    Winston-Salem, North Carolina, 27157, United States

  • Yale University School of Medecine

    New Haven, Connecticut, 06510, United States

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