Participant FAQs

Hundreds of people across the UK are taking part in MND-SMART. The trial has been designed so that most people with MND will be eligible.

People who are pregnant or breast-feeding and those who are already part of another drug trial will not be able to take part. There are some other criteria too; the trial team will discuss these with you

You can still take part in MND-SMART if you are taking Riluzole, receiving breathing support, or are on non-invasive ventilation.

No. How long you have been diagnosed with MND doesn’t affect whether you can join the trial.

No. MND-SMART is only open to people with MND living and receiving their MND treatment in the UK.

Yes, you can register interest at any point.

MND-SMART is intended to be a long-term study. For the trial to be successful, we need people with MND to continue to join the trial in the coming months and years so that we can continue to test new drugs.

We will recruit people as quickly as we can however this is limited by the capacity of the teams in each trial centre to ensure participant safety and a high standard of patient care

No, you will not be paid for taking part. We can help towards travel expenses. Please speak to your local trial centre for information.

We are opening sites as rapidly as possible across the UK. When we open a new site, we will contact those who live near the site who have sure you have registered their interest on our website.

We are very sorry that your MND is progressing and we are doing everything we can to ensure as many people as possible can enter the trial across the UK. Unfortunately willingness to travel, relocate or stage of disease progression will not influence whether you are invited to join the trial or not.

You will take a medicine once a day. We will ask you to keep a diary to record when you took the drug, and any side-effects you experienced. You will also need to attend appointments. Some appointments will be at your trial centre and the rest can be done over telephone or video conference.
 

After your baseline appointment there are some weekly appointments to review safety and increase your trial dose (if applicable), but later appointments are only every 2 months. Some appointments will be at the participating trial centre nearest to you, and the rest can take place over the phone or by secure video calls.

Your involvement could be anything from a few weeks up to a maximum of about 7 years. This depends on when you join the study and whether the trial data shows if a drug has any benefit for people living with MND.

The benefit of this is that participants won’t take ineffective drugs for longer than they need to.

The tests and assessments vary between different appointments. These will include questionnaires about physical functioning, your mental health and your MND, blood tests, blood pressure, an ECG (heart trace), breathing tests, a cognitive (thinking, learning and memory) assessment, and a pregnancy test if relevant.

Yes, we can pay some expenses for you to travel to your nearest trial centre for appointments. Additional grants may be available depending on where you live; please speak to your local trial centre for further information.

People are assigned to a trial drug, or to the placebo, at random by a computer program. No-one involved in the trial can influence whether a participant receives a trial drug or placebo. This makes sure the results of the trial are not biased.

No. This is a double-blind study, which means that neither the study team or trial participant know who is taking an active trial drug and who is taking a placebo. Drugs are specially packaged and labelled without their name to make sure that nobody involved in the research finds out who is taking what. If your doctor needs to know what trial drug you are taking, in the event of a medical emergency, they can find out.

Please see information on our Trial Drugs page to find out what drugs are currently being tested.

We have a rigorous pipeline for drug selection, which includes automated searching of hundreds of scientific publications written by doctors and scientists worldwide, as well as laboratory studies that test the effects of drugs on human brain cells that we grow from stem cells donated by people living with MND. All the evidence is scrutinised by our drug selection committee, which includes clinical experts.

MND-SMART is designed to run continuously for years to come. To help select future drugs we will trial, we will repeat the process of reviewing published information from MND and neurodegenerative disease studies at regular intervals.

We include all published studies on stem cells in our unbiased pipeline for drug choice. We are also working on gathering further information on potential drugs by testing them in our labs on stem cells derived from people with MND.

If, in the future, treatment with stem cells show the most promise for beneficial effects in MND, according to the scientific evidence, we may include them in MND-SMART.

No. It’s really important that participants continue to take the medication that they have been assigned to. We need participants to continue taking the current trial drugs until we have enough data to draw conclusions about whether the drugs are working. We will make the findings known as soon as they are available.

No, we always planned to test more drugs as part of MND-SMART. We select the drugs to be tested in the trial according to the best available evidence at the time.

We need to continue gathering data to decide if the other drugs being tested are working. We will make the findings known as soon as they are available.

No. We select the drugs to be tested in the trial according to the best available evidence at the time. We have a rigorous pipeline for drug selection, which includes automated searching of hundreds of scientific publications written by doctors and scientists worldwide, as well as laboratory studies that test the effects of drugs on human brain cells that we grow from stem cells donated by people living with MND. All the evidence is scrutinised by our drug selection committee, which includes clinical experts.

We will work hard to make this happen as quickly as possible. If we find a drug leads to meaningful changes when we analyse the trial data, we will apply to the early access to medicines scheme. This is run by the Medicines and Healthcare Product Regulatory Agency (MHRA) who approve the use of all drugs in the UK. They will review the trial results and decide if, and when, the drug could be made available.

Please remember it will take many years to collect all of the data required to complete the final trial analysis required for this application.