A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients with Wilson's Disease
This research is testing a new treatment called VTX-801 for adults who have Wilson's disease. This is an early study, meaning it's one of the first times this medicine is being given to people. The main goal is to check how safe VTX-801 is and if people can tolerate it well. Researchers will closely monitor for any side effects, and they'll also look at changes in body measurements, blood tests, brain scans, and tummy scans. A key part of the study is to see how the medicine affects copper levels in the body, which is important for Wilson's disease, and whether the body's immune system reacts to the new medicine. Participants will be followed for five years to understand the long-term effects.
At a glance
What is this study about?
This study is looking into a new treatment called VTX-801 for people who have Wilson's disease. Wilson's disease is a rare condition where too much copper builds up in the body, which can cause serious health problems. The new medicine, VTX-801, aims to help manage this copper build-up. This type of research is in its early stages (called Phase I and Phase II), which means it's one of the first times doctors are giving this specific medicine to people. The study will look at different doses of the medicine to find the safest and most effective amount.
The main purpose of this study is to check how safe VTX-801 is and whether people taking it experience any side effects. Doctors will carefully watch participants for any health changes and record them. They will also do various tests, like blood samples, check vital signs such as blood pressure and heart rate, perform heart tracings (ECG), and do scans of the brain and tummy. These tests will help them understand how the body reacts to the new medicine.
Beyond safety, the study will also measure other important things. Researchers will look at copper levels in the blood and urine, and a protein called ceruloplasmin, which is involved in copper transport. They also want to see if VTX-801 helps improve the condition in a noticeable way and if the body's defence system (immune system) reacts positively or negatively to the medicine. Participants in this study will be followed for five years to see the long-term effects of the treatment.
Key takeaways
- Tests a new medicine (VTX-801) for Wilson's disease.
- First time the medicine is given to people (early stage).
- Main goals: check safety and how well people tolerate it.
- Will monitor copper levels and body's reaction to medicine.
- Participants will be followed for five years.
- Only for adults (18+) with Wilson's disease.
Who may be eligible?
This study is specifically for adults who have been diagnosed with Wilson's disease. You must be at least 18 years old to be considered for participation. There is no upper age limit, meaning older adults with Wilson's disease can also potentially join.
Because this is an early-stage study, there will be strict rules about who can and cannot take part. These rules are in place to make sure the study is as safe as possible for everyone involved and that the results are clear. For example, people with other serious health conditions or who are taking certain medications might not be able to join.
If you have Wilson's disease and are over 18, and you are interested in learning more, you should speak to your doctor or the study team. They will be able to go through your full medical history and explain if you meet all the specific requirements to participate.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have a confirmed diagnosis of Wilson's disease?
- Are you able to attend appointments for five years?
- Are you willing to have blood tests, scans, and ECGs?
- Are you comfortable with receiving a new, untested medicine?
What does participation involve?
If you decide to take part in this study, you would receive a single dose of the new medicine called VTX-801. This is a one-time treatment, but your health will be monitored very closely for a long time afterwards. You would have several visits to the study clinic, especially in the beginning, for check-ups and tests. These tests include physical examinations, blood and urine samples, checks of your heart via ECG, and scans of your brain and tummy (MRI).
You would also be followed up for a full five years after receiving the dose. During this time, you would have regular appointments to ensure the medicine is safe, to check for any long-term effects, and to see if your Wilson's disease is responding well to the treatment. The total duration of your active involvement with clinic visits and follow-ups would be five years, with more frequent visits initially.
Potential risks and benefits
Locations (2)
- —UnverifiedGermany
- —UnverifiedDenmark
Common questions
What is Wilson's disease?
Wilson's disease is a genetic condition where your body can't remove extra copper, causing it to build up in organs like the liver and brain.
Is VTX-801 a cure for Wilson's disease?
We don't know yet. This study is testing if it's safe and if it helps to reduce copper levels, but it's not known if it's a cure.
How long will I be in the study?
You will be followed by the study team for five years after you receive the single dose of the medicine.
Will I have to pay to join the study?
No, you will not have to pay to receive the study treatment or for the study-related visits and tests.
What if I get side effects?
The study team will be watching you very carefully for any side effects and will provide care and support if you experience any.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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