A Clinical Trial of Treatment to Slow Disease Progression in Children and Adults with Wolfram.
The two possible treatments are:
A- A medicine used in children and adults
This treatment is for 3 years, until final assessments to see if it works. These are tablets.
B- Placebo treatment
This is also for 3 years. The dummy tablets look /taste identical to the real medicine but do not contain the study medication. The patient and doctor will not know which treatment is which.
This study is in Birmingham, UK; Spain; France; and Poland. 70 people will take part. 2 people will have the medicine to every 1 the placebo, decided by chance.
First visit, up to 28 days before starting
- Ask consent to take part
- Baseline tests and brain scan
- Check person is eligible to take part
Second visit, randomise to medicine or placebo
- Start treatment Stage 3
Follow-up visit at 6 weeks
- Visit for safety checks Stage 4
Visits at 6, 12, 18, 24, 36 months
- Safety checks, blood tests, eye tests
- MRI brain scans at 12, 24, 36 months
Final visit after treatment finished
- Final safety checks
This trial will tell us if this medicine is SAFE and EFFECTIVE in Wolfram. If these are shown, then doctors may prescribe this medicine for almost everyone with Wolfram in any country.
5. Am I eligible to take part?
For the US trial, Prof Urano is currently recruiting patients in the US.
For the European trial, recruitment will start in the UK in Autumn 2017; and in Spain, France and Poland, in Spring 2018.
People will have checks to make sure they have Wolfram syndrome; enough vision at the start of the study to be able to assess the effects of treatment; and safety checks.
6. Possible benefits of taking part
Taking part in a clinical trial can be helpful if the trial medicine works to slow down the progress of the disease. However there is no guarantee that this will happen. There may be benefits in being seen by the study team regularly, as they will monitor the Wolfram disease carefully and treat any complications if they happen.
7. Possible risks of taking part
Any medicine in a clinical trial may not work, and not slow down the disease; and every medicine has side effects. Sometimes these side effects may be serious or life threatening. Before deciding whether or not to take part, always read the participant information sheets carefully, and ask about possible side effects.
8. How will we know if a medicine works in Wolfram?
We asked patient support groups in the UK, USA, Spain, and France, to advise us what are the most important outcome measures for trials of new treatments. Each group ranked vision as the most important. This means that any treatment must slow down or stop the worsening of vision.
Prof Tamara Hershey has shown that in people with Wolfram, parts of the brain slowly get smaller with age. This is related to problems with balance. With her kind help, we are using brain scans, to check that any treatment can slow down or stop parts of the brain getting smaller.
In Wolfram, vision gets worse, and parts of the brain get smaller, over years rather than months. In order to show that a treatment works, we need to give the treatment for several years. This is why the European treatment trial involves giving people the medicine for 3 years.
If it is clear that a medicine in a clinical trial is working, it is possible to stop the trial early and give everyone the medicine.
9. Can I take these medicines without taking part in a trial?
The medicines being used in the clinical trials are not licensed for use in Wolfram. This means that doctors are not allowed to prescribe them to treat Wolfram syndrome. The reason is that no- one has done studies to show they are safe and to show they work in Wolfram. If a person with Wolfram took one of these medicines outside a trial, and had a serious side effect, the clinical trial would automatically be stopped. If this were to happen, we would never know if these medicines may work in Wolfram.
10. Future plans for studies
If Dr Urano’s safety study of his medicine shows that it is safe, he will then apply for a Phase II study to see if it works in Wolfram. The design of this study will be similar to the European trial described above.
We are also seeking European Medicines Agency advice about the design of a Phase III clinical trial comparing both the medicine in the US trial and the medicine in the European trial, in combination in Wolfram. If this is funded, it will start after the current studies have finished. It will recruit up to 140 people with Wolfram worldwide.