Leber's Hereditary Optic Neuropathy Idebenone Trial
THIS TRIAL IS ONLY OPEN TO LHON PATIENTS WHO LIVE IN THE UK OR GERMANYClick on the questions below to bring you to the answers further down the page. Links beside each heading will return you to the top of the page.
- What is idebenone (SNT-MC17)?
- What are the aims of this LHON trial?
- Who will be eligible to take part in the trial?
- What will taking part in the trial involve?
- What tests will be carried out?
- How will I get to the trial centre?
- What are the possible risks of taking part?
- What are the possible benefits of taking part?
- Do you want more information?
What is idebenone (SNT-MC17)?
Idebenone (SNT-MC17) is a drug with “anti-oxidant” properties which means that it has the ability to protect cells from damage when the mitochondria stop working properly. Moreover, idebenone (SNT-MC17) can help sick mitochondria produce more energy.
What are the aims of this LHON trial?
There is no available treatment for LHON and there is an urgent need to find one. Since mitochondria do not function properly in LHON, we are interested in testing idebenone’s (SNT-MC17) potential beneficial effect in this condition. We want to know whether giving idebenone (SNT-MC17) to patients affected with LHON will enable them to see better.
Who will be eligible to take part in the trial?
- We will be recruiting LHON patients who have lost vision for less than 5 years.
- Patients must have one of the three most common genetic LHON mutations (3460, 11778 and 14484). This will involve a genetic test if it has not been done already by your local specialist.
- Patients must be 14 years of age or older but must be below 65.
- Patients must not be pregnant or breast feeding.
- These are the main points. Other inclusion and exclusion criteria will be checked before deciding whether patients are eligible to enter the trial.
What will taking part in the trial involve?
Because of regulatory requirements the study needs to be “placebo-controlled” if any beneficial effect is to be proven. Therefore, two thirds of the patients will receive the study medication idebenone (SNT-MC17) while the other one third will receive look-alike but inactive placebo “dummy” tablets.
Neither the patient nor the study doctors will know who will receive idebenone (SNT-MC17) and who will receive the placebo.
All patients will take the study tablets for 24 weeks and will have regular follow-ups with an eye doctor.
Patients who enter this clinical trial will have to attend the trial centre in Newcastle upon Tyne for a total of 6 visits over a period of 28 to 32 weeks.
If idebenone (SNT-MC17) is shown to have a beneficial effect, patients finishing this study will be invited to join an extension study in which there is no placebo and everyone will be taking idebenone (SNT-MC17).
What tests will be carried out?
The study doctor will give you a medical check-up at each visit and there will be some straightforward questionnaires to fill in.
In addition, the following tests will also be carried out:
- Physical examination
- Heart tracing (ECG), blood and urine samples
- Eye Examination
- Vision tests
Not all of these tests will be done at each clinic visit. Taking a small blood sample will be the only procedure that might cause a little discomfort.
How will I get to the trial centre?
The trial centre for the UK is based at the Royal Victoria Infirmary (RVI) in Newcastle upon Tyne. You will be seen by the study doctor in the Clinical Research Facility first, before going to the Eye department for the remaining tests. Details of how to get there are available on the directions page. You will receive a travel allowance to cover the travel expenses and accommodation if needed.
What are the possible risks of taking part?
All the tests that are going to be carried out in this trial are used frequently by eye specialists and are not harmful.
Idebenone (SNT-MC17) has been studied in other diseases in large groups of patients. It is well tolerated and has demonstrated a low level of side effects.
What are the possible benefits of taking part?
Patients taking part in this trial will receive a detailed medical check-up. We do not know yet whether SNT-MC17/idebenone is going to be an effective treatment for LHON. However, even if SNT-MC17/idebenone is proven not to be useful, the knowledge gained from this research study will further our understanding of this condition and therefore is likely to benefit LHON families in the long term. If the results from this trial are positive, e.g. the damage to the optic nerve can be limited such that vision in affected patients is preserved or improved, idebenone (SNT-MC17) may get regulatory approval as a prescription drug in LHON.
Do you want more information?
Please get in touch with us using the contact details provided in this website if you would like to receive an information sheet or discuss some specific points about this trial with a member of our clinical research team.
All the information about your participation in this trial will be kept strictly confidential