MND-SMART Clinical Trial

About the project

MND-SMART is innovative drugs trial designed to speed up the time it takes to find medicines that can slow, stop, or reverse the progression of, MND. It is designed to include as many people with MND as possible, regardless of how the disease or current treatments affect them.

MND-SMART stands for Motor Neurone Disease – Systematic Multi-arm Adaptive Randomisation Trial. 

Multi-arm means that, unlike typical clinical trials which test a single treatment, MND-SMART will test more than one at the same time. Trial participants taking the different treatments will be compared with a single group who receive a dummy drug, called a placebo. This means that people in MND-SMART are more likely to receive an active treatment when compared to standard clinical trials where half of participants receive the placebo and half the active treatment.

The trial is also adaptive so that researchers can change the drugs being tested according to emerging results. This means that new medicines can be added once a trial has started, while treatments that do not prove effective can be dropped. This is a phase 2 / 3 trial which means if drugs appear to be effective, they will seamlessly transition into phase 3 without the need for additional permissions or trial participants needing to complete a phase 2 trial and join a separate phase 3 trial. 

The range of experts involved in designing the trial are from many different clinical and academic backgrounds including MND consultants and nurses, statisticians and lab scientists.

We’ve involved and listened to people with MND throughout the trial design process and so:

  • we’ve made the clinical trial design that is open to as many people with MND as possible with very few exclusion criteria
  • the study drugs are being given as liquids which will make them as easy as possible for people to swallow
  • we’ve introduced the option of video calls for some appointments to reduce the number of times people have to travel to a Clinic
  • we’ve made the language we’ve used in the trial materials as easy to understand as possible

The drugs

The trial will initially test medicines that are already approved as treatments for other diseases. This means there is a reduced risk of adverse effects in trial participants. Repurposing drugs in this way also avoids some of the lengthy approvals processes needed for new drugs and could cut years off the time taken for the medications to become more widely available. MND-SMART is a long-term study that will test medications for many years to come. 

To find out more about the project visit



Euan MacDonald Centre for MND Research, MND Scotland and My Name'5 Doddie Foundation alongside individual donors.


Primary location


Principal Investigator

Other people involved

Dr Suvankar Pal, Edinburgh Medical School

Judy Newton, MND Nurse Consultant

Dr Rachel Dakin, Trial Manager