Participant FAQs

Participant FAQs

Find the answers to frequently asked questions about the trial.


Our FAQs are split under three headings - Taking part in MND-SMART, Covid-19 update and trial design.

Taking part in MND-SMART

Hundreds of people across the UK will be able to take part at participating trial centres. 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, have trouble swallowing or have a feeding tube.
 

Unfortunately Covid-19 has impacted on the pace that we've been able to open sites across the UK. The pandemic has meant that many research teams do not currently have the staff available to work on MND-SMART as research teams in particular have been redeployed to wards and clinical areas.

There are many steps to opening a new site. These include internal department checks, legal agreements, financial arrangements, staff training, approval from local governing committees and other paperwork. All of these procedures must be in place before a site can open and they ensure that a trial centre is fully prepared to safely and legally recruit trial participants at their site.

Unfortunately it may be that a trial centre does not open near you. Participants in clinical trials need to be followed up regularly to assess if potential treatments are working, and also so they can be monitored closely for safety. Due to this, trial centres are unable to assess people who live a long distance away and each centre has a certain capacity of how many participants they can recruit and monitor.

Although this may disappoint some people that they may not be able to take part in MND-SMART, this is an exciting time of rapid progression across all MND research in the UK. A number of different clinical trials are planned and good progress is being made in research labs worldwide to understand more about the disease and devise potential new treatments. Results from all of these studies, including MND-SMART will, over time, help provide information about the safety and effectiveness of drugs and therefore have the potential to benefit everyone living with MND.

You can find out which MND-SMART centres are open on the Trial Centres page of our website.

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.

We also strongly discourage people from considering moving to the UK to try and take part in the study. This would interrupt your clinical care. Also we cannot guarantee that you would be able to take part in the trial as a large number of people in the UK have already registered interest in taking part.
 

You will take a liquid 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.

At the appointments we will ask you about the progress of your MND, answer any questions you have and take you through a set of questionnaires and physical assessments.
 

There are several weekly appointments early on whilst the dose of medicine is increased safely, 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. At these appointments we will ask you about the progress of your MND, answer any questions you have and take you through a set of questionnaires and physical assessments.

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 your mental health and your MND, blood tests, an ECG (heart trace), breathing tests, a cognitive (thinking, learning and memory) assessment, and a pregnancy test if relevant. Your physical function will also be assessed against the ALS Functional Rating Scale. This allows the research team to evaluate the progress of your MND by reviewing things such as your speech, breathing, handwriting and walking.
 

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.
 

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.
 

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 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.

Please remember that MND-SMART is a research study and the drugs being tested will not cure MND, may cause side effects, and participants have a 1 in 3 chance of being allocated to may receive a placebo (dummy drug). As we continue to roll out the trial across the country, please know that we will share any reliable findings as soon as we can.

 

Covid-19 update

Since the Covid-19 pandemic began in the UK in March 2020, we have continued to work with other research teams across the country to prepare for new sites to open as soon as possible. This means that we now (March 2021), have seven trial centres open.

Understandably this work has been hindered by the pandemic with research staff redeployed to the NHS front lines and restrictions on face-to-face appointments for initial trial assessments.

This means our original timescales for opening new trial sites have been affected. We are committed to opening sites as soon as we can though and continue to do all we can within the restrictions to make this happen.

Positively, unlike many other clinical trials, since emerging from lockdown in August 2020, we have continued to recruit and follow up participants in sites that are already open in line with support from our regulators and delivery of essential healthcare.

The design of the MND-SMART trial is what has allowed us to continue to run the trial and effectively follow up participants. From the start of the trial we have had approval from the trial regulators to do video-call appointments and courier drugs to participants so no time was spent seeking new permissions.

 

 

Trial design

Please visit the Trial Drugs page of our website for full information on the drugs being tested, how they were selected and how we will choose new drugs to add to the trial.
 

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.
 

MND-SMART is an adaptive clinical trial. This means the results gathered during the study will be reviewed at regular intervals whilst participants are still taking the study drugs. Traditional clinical trials wait until the end of a 2 or 3 year study before they confirm their findings.

To prove whether the drugs are working or not, we need to have collected enough scientific data for review. The first data measurement (‘interim analysis’) will take place when data from 50 people has been gathered for a minimum of 6 months to have a true picture of whether a drug is working or not.

We are currently testing two drugs and a placebo so once 150 people have been recruited to the trial we can review our data 6 months after that. We predict that this review point will be in late 2021.

If at this review point a medicine does not appear to be working, it may be removed from the trial. If the data suggests that a medicine is having a positive effect on those taking it, or the results are inconclusive, the medicine will continue to be tested and the data reviewed again after a set period of time.
 

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.
 

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.

We need a placebo group so that we can prove any changes we see in the trial participants are due to the active drugs and not any other factors.

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