RecruitingPHASE2, PHASE3INTERVENTIONAL

Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial

The MND-SMART study is investigating ways to slow down Motor Neurone Disease (MND) and improve survival for people living with the condition. It's a special type of study that allows researchers to test several different medicines at the same time and change which drugs are being studied based on the results they get. This means that if a drug isn't working, it can be stopped, and new, promising treatments can be quickly added. Currently, the study is looking at Amantadine and Tacrolimus, comparing them to dummy treatments (placebos). The drugs being tested are already approved for other health conditions. The aim is to find effective treatments for MND more quickly and efficiently.

At a glance

Status

Recruiting

Phase

PHASE2, PHASE3

Sponsor

University of Edinburgh

Enrolment target

1,150

Start

27 Feb 2020

Estimated completion

01 Dec 2030

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

What is this study about?

MND-SMART is a really important research study looking into new ways to treat Motor Neurone Disease (MND), also known as Amyotrophic Lateral Sclerosis or ALS. Doctors and scientists want to find medicines that can slow down how quickly MND progresses and help people live longer and healthier lives. Currently, there aren't many treatments for MND, so this study aims to find more options.

This study is quite clever because it's designed to be very flexible. It's like a scientific 'try-it-out' system where several different medicines can be tested at once. If a medicine isn't showing much benefit, the study can quickly stop using it. On the flip side, if scientists discover a new promising drug, they can add it to the study relatively fast. This 'adaptive' approach means they can evaluate many potential treatments efficiently over time, helping to get effective medicines to people sooner.

To decide which medicines to test, a group of independent experts carefully reviewed all the existing information about MND, including past studies and lab research. They picked drugs based on how they might work, how safe they are, and the quality of previous research. The study continues to look at new scientific evidence and discoveries to find the best possible drugs to investigate.

Key takeaways

MND-SMART is a flexible study looking for new MND treatments.
It tests several medicines at once, including Amantadine and Tacrolimus.
Drugs that don't work are removed, and new ones can be added.
Experts carefully choose which drugs to investigate.
Participation involves potential new treatments but also the possibility of a placebo.
The study aims to find effective MND treatments more quickly.

Who may be eligible?

To join the MND-SMART study, you must have a confirmed diagnosis of Motor Neurone Disease. You need to be over 18 years old and willing to follow the study's instructions, including filling out questionnaires. If you're a woman who could become pregnant, you'll need to have a negative pregnancy test and agree to use effective contraception during and for a short time after the study treatment. Men who could father children also need to use contraception.

There are some reasons why you might not be able to join. For example, if you have a certain type of dementia (Frontotemporal Dementia) or a serious mental health condition that makes it hard to understand the study, you wouldn't be able to participate. You also can't be pregnant or breastfeeding, or have certain other medical conditions or take other specific investigational medicines. The study team will carefully check all your medical information to make sure it's safe for you to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Motor Neurone Disease?
Am I 18 years old or older?
Am I willing to follow study instructions and use contraception if needed?
Am I pregnant or breastfeeding? (If yes, I might not be able to join).
Do I have a serious mental health condition or certain other medical issues mentioned by my doctor?

Answer every question to see your result.

What does participation involve?

The information provided does not detail the specifics of visits, assessments, or total duration. However, generally, taking part in a clinical trial like MND-SMART would involve regular visits to a study clinic. During these visits, you might have check-ups, answer questions about how you're feeling, and have tests like blood samples taken. You would be given a study medicine (either the active drug or a dummy pill called a placebo) to take regularly. The study team would closely monitor your health and how the medicine is affecting you. You would have follow-up appointments after you finish taking the study medicine to see how you are doing.

Potential risks and benefits

Potential benefits of joining could include receiving a promising new treatment for your MND that might slow its progression or improve your symptoms, and contributing to important research that could help others with MND in the future. However, there's no guarantee the treatment will work for you, and you might receive a placebo. All medicines carry potential risks, including side effects, which the study team will monitor closely and discuss with you. You have the right to withdraw from the study at any time without giving a reason, and this will not affect your usual medical care.

Locations (22)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Southern Health and Social Care Trust, Craigavon Area Hospital
Verified postcode
Portadown, United Kingdom· Recruiting
Aberdeen Royal Infirmary
Verified postcode
Aberdeen, United Kingdom· Recruiting
University Hospitals of Birmingham NHS Foundation Trust
Verified postcode
Birmingham, United Kingdom· Recruiting
University Hospitals Sussex NHS Foundation Trust
Verified postcode
Brighton, United Kingdom· Recruiting
West Suffolk NHS Foundation Trust
Verified postcode
Bury St Edmunds, United Kingdom· Recruiting
Cambridge University Hospitals NHS Foundation Trust
Verified postcode
Cambridge, United Kingdom· Recruiting
Cardiff and Vale University Local Health Board
Verified postcode
Cardiff, United Kingdom· Recruiting
Clinical Research Centre , Ninewells Hospital
Verified postcode
Dundee, United Kingdom· Recruiting
Anne Rowling Regenerative Neurology Clinic
Verified postcode
Edinburgh, United Kingdom· Recruiting
Royal Devon and Exeter Hospital
Verified postcode
Exeter, United Kingdom· Recruiting
Queen Elizabeth University Hospital Clinical Research Facility
Verified postcode
Glasgow, United Kingdom· Recruiting
NHS Highland Clinical Research Facility, Raigmore Hospital
Verified postcode
Inverness, United Kingdom· Recruiting

Common questions

What is MND-SMART?

It's a research study testing different medicines to see if they can slow down Motor Neurone Disease (MND) and help people live longer.

Why is it called 'adaptive'?

It means the study can change which medicines are being tested over time, dropping ones that don't work and adding new promising ones quickly.

What kind of medicines are being tested?

Currently, the study is looking at Amantadine and Tacrolimus, alongside dummy treatments (placebos). These drugs are already approved for other health problems.

Will I definitely get an active drug?

Participants are randomly given either an active drug or a placebo (dummy treatment) to fairly compare how each works.

What happens if a medicine doesn't work?

If a medicine is found not to be helpful, the study can stop using it, and focus on other potential treatments.

How to find out more

Professor Chandran

siddharthan.chandran@ed.ac.uk 0131 465 9612 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.