Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Can a One-Time gene fix cure wilson disease?

NCT ID NCT06663878

Cure Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 12, 2026 · Last updated Aug 13, 2026 · Updated 1 time

Summary

This trial tests an experimental gene therapy called MWAV201 in adults with Wilson disease, a genetic condition that causes copper to build up in the body. The treatment uses a harmless virus to deliver a corrected gene, potentially restoring the body's ability to remove copper. The study focuses on safety and tolerability, while also measuring copper levels in the blood and urine to see if the therapy is working.

What this could mean

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

Active substance
MWAV201, a nonreplicating recombinant gene transfer vector
What this could lead to
If successful, this gene therapy could provide a one-time treatment that corrects the underlying genetic defect in Wilson disease, potentially reducing or eliminating the need for lifelong medication.
What could go wrong
This is an early-stage, dose-escalation study with only 9 participants, so safety and efficacy are not yet established. Gene therapy carries risks such as immune reactions or unintended effects, and the treatment may not work for all patients.

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

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 9 people

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

Started

Oct 2024

Expected to finish

May 2031

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

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.

Inclusion Criteria: * Male or female aged 18 and 65 years inclusive; * Confirmed diagnosis of Wilson disease; * Low copper diet and standardized medication for ≥ 1 year; * Stable Wilson disease for ≥ 1 year; * Able to understand and willing to follow study procedures. Exclusion Criteria: * Significant hepatic inflammation as evidenced by liver function test. * Liver biopsy or liver stiffness measurement show progressive liver fibrosis. * Laboratory tests or clinical symptoms indicate decreased liver reserve function. * Other chronic liver disease (such as hepatitis B). * Any signs of decompensated liver function (such as ascites). * History of liver transplant or plan to receive liver transplant. * Other diseases with clinical significance, such as cardiovascular and cerebrovascular diseases, kidney diseases, respiratory system diseases, neurological diseases, mental illnesses, active infections, etc. * Body Mass Index ≥ 30 kg/m2. * Other conditions that, in the Investigator's opinion, may not be suitable for the subject to be enrolled.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Wilson disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

  • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

    Shanghai, Shanghai Municipality, 200092, China

More trials for these conditions

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