A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

To join this study, you must be at least 18 years old and have a confirmed diagnosis of Wilson disease, usually proven by genetic tests. You should also be managing your Wilson disease with regular copper-removing medicines or zinc therapy for at least two months without any recent changes to your medication or diet. You'll also need to be willing to follow all the study instructions, including regular blood tests and providing urine samples.

There are also reasons why someone might not be able to join. For example, if you've already had a liver transplant or if your liver disease is very severe. Other reasons could include having serious heart or kidney problems, certain mental health conditions, or if you've had a strong allergic reaction to similar medicines in the past. If you have a history of not consistently taking your Wilson disease medication, or if you've been in another similar gene therapy study, you might also not be eligible.

If you decide to take part in this study, you will receive either UX701 or continue with your standard care. Everyone who receives UX701 will also be given other medications to help prepare your body for the treatment and to prevent side effects. These include medicines called premedication, corticosteroids (like a type of steroid), and other treatments to help your immune system.

Throughout the study, you'll have regular visits to the clinic. These visits will involve various checks, including frequent blood tests, collecting all your urine over a 24-hour period, and completing questionnaires about how you're feeling and your quality of life. The study involves a long-term follow-up period, meaning that after you receive the treatment, doctors will continue to monitor your health and how well the treatment is working for at least five years. The total time you'll be involved in the study will vary, but expect a commitment over several years due to these follow-up checks.