Frequently asked FAQs

What can you tell me about the drugs being tested?

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

When will you have results and know if the drugs are working?

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.

If a drug is found to have a positive effect, how long will it take to become available to everyone?

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.

When are you opening in [place name]?

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.

My MND is progressing and I am deperate to join the trial. Can I get an appointment soon?

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.

For more frequently asked questions and answers, visit our Participant FAQs page.

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