Can a One-Time gene fix cure wilson disease?
NCT ID NCT06663878
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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.
- Hidden heart risks in Wilson's disease: a new scan could spot them early
- Can a single infusion rewrite the genetic code behind wilson disease?
- Newborn screening study aims to catch rare diseases at birth
- Building a database to unlock wilson disease mysteries
- New Free-Breathing MRI could make liver scans easier for kids
- One-Time gene shot could free wilson patients from daily pills