Wilson’s disease is a rare, inherited liver condition. People with Wilson’s disease have to take medicine for their whole life to stop copper building up in their bodies.
If you’ve been diagnosed with Wilson’s Disease and are over 18, you might be able to join a new clinical trial. This is a chance to help test a possible new treatment. And help researchers learn more about this rare condition.
Who can take part?
You can join if:
- You are 18 or older.
- You have been diagnosed with Wilson’s Disease.
- You take medicine for your Wilson’s disease at least twice a day and have done so for at least 3 months.
- You take no more than 1200 mg a day of a medicine called a chelator (like Penicillamine or Trientine).
- If you’re sexually active, you use a suitable type of contraception.
What is the study testing?
The study will look at a new version of the medicine Trientine. The new medicine is taken just once a day.
The researchers want to know if people with Wilson’s disease prefer this treatment to their usual medicine.
What will I have to do if I take part?
The whole study lasts 8 weeks. You will be asked to stop taking your usual medicine for 4 weeks. During this time you will take the new medicine instead.
You will then go back to your usual medicine and the study team will follow you for another 4 weeks.
You will also:
- Be asked to fill in regular questionnaires
- Have a phone call with a doctor every 2 weeks
- Have 3 in person study appointments at a location you choose.
The 3 study appointments will take up to 2 hours. You do not have to go to hospital for these. They could be at your home or at a clinic near where you live or work.
During the study you will have:
- Talks and checks
You will be asked about your health and any medicines you take. You can also ask any questions you have about the trial and about the risks and benefits of taking part.
- Safety Blood tests
You will have regular blood tests to check the safety of the new treatment.
- Copper blood test
You will have a blood test to see how well the new medicine removes copper from your body.
- Pregnancy test
If there is any chance you could become pregnant you will have pregnancy tests throughout the study for safety reasons.
What are the risks and benefits of taking part?
The risks of taking part are thought to be low. The side effects of the new medicine are similar to those that you may already have with your current treatment.
There is no benefit to your medical condition, but you will be helping researchers to improve treatment for Wilson’s disease in the future.
It will not cost you anything to join the study. You will be paid compensation for taking part. You will also be able to claim for any expenses such as travel costs.
Find out more
If you are interested in joining the trial, or you would like to find out more, it you can:
- call 01332 429760 (and select option 3)
- email information@thevctc.com.
You can also find out more about Wilson’s disease here.
Who is running the trial?
The new medicine is being developed by Orphalan. The research is being done by VCTC. They are a clinical trials site that aims to make it easier for people to take part in research.
