Wilson Disease: UX701 Gene Therapy Study

We are evaluating a new gene therapy for adults with Wilson disease to see if it safely helps manage copper levels in the body. The study will also look at how it affects the need for current medications and overall liver health.

1-2 yearsSafety phase (I)Efficacy phase (II)Post-Trial Drug AccessNo PlaceboCost ReimbursementInvestigational MedicinesOralInjectionHepatologyInfectious DiseasesInternal Medicine

What diseases are being studied?

Health conditions and diseases that the clinical trial is designed to study and treat.

Who can participate in the clinical trial?

These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..

What medicines are being studied?

Medicines that are officially approved for use in the European Union and considered safe by EMA. These medicines are already on the market and may be tested for new conditions or populations.

Prednisolon Stada
Prednisolon Stada is a steroid medicine used to reduce inflammation and treat conditions like allergies, asthma, and certain immune-related disorders.
Tacrolimus
Tacrolimus is a medicine that suppresses the immune system to help prevent organ transplant rejection and to treat severe eczema.

What active substances are being studied?

Active substances that are officially approved for use in the European Union and considered safe by EMA. These substances are already on the market and may be tested for new conditions or populations.

Eculizumab
Eculizumab is a substance that blocks part of the immune system to prevent blood cell destruction and reduce attacks in certain rare immune disorders.
Prednisolone
Prednisolone is a steroid substance that reduces inflammation and suppresses an overactive immune response.
Rituximab
Rituximab is a substance that targets and reduces B cells, a type of white blood cell, to treat some blood cancers and autoimmune conditions.

What experimental substances are being tested?

Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.

Adeno-Associated Viral Vector Serotype 9 Encoding Human Atp7b

What similar medicines might you know?

Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.

Adelcort®
Adoport
Adport

Where is the clinical trial being conducted?

Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.

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Aarhus Universitetshospital
Department Hepato- and Gastroenterology
Århus, Denmark
Centre Hospitalier Universitaire De Toulouse
Service de Neurologie
Blagnac, France
Fondation A De Rothschild
Service de Neurologie
Le Pré-Saint-Gervais, France
Sponsor: Ultragenyx Pharmaceutical Inc.
Last updated: Nov 26, 2025

Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.