RecruitingPhase IIInterventional

EXPErimental medicine Route To Success in Amyotrophic Lateral Sclerosis (EXPERTS-ALS)

Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease. It causes muscle weakness that worsens over time, and current treatment options are very limited. The EXPERTS-ALS study is testing several well-known drugs already used for other health conditions to see if any can help people with ALS. In this study, researchers measure a nerve protein in the blood called neurofilament light chain (NFL). High levels of NFL suggest nerves are under stress. If a drug lowers these levels, it could mean it helps protect nerve cells. This is an 'adaptive' study, meaning scientists can quickly drop drugs that show little promise and test new ones. There is no placebo (dummy pill), so everyone who joins will take an active medication for up to six months while being closely monitored by doctors.

At a glance

Status

Recruiting

Phase

Phase II

Sponsor

Sheffield Teaching Hospitals NHS Foundation Trust

Enrolment target

700

Start

18 Nov 2024

Estimated completion

31 Mar 2028

Amyotrophic lateral sclerosis (ALS)

What is this study about?

Amyotrophic lateral sclerosis (ALS) is a serious condition that affects the nerves responsible for movement. Over time, it leads to muscle weakness, making everyday tasks like walking, talking, swallowing, and breathing harder. Right now, there is no cure, and finding new treatments has historically taken a very long time. The EXPERTS-ALS trial aims to speed up this search by screening several potential medications quickly and safely in people living with the condition.

Rather than inventing brand-new treatments from scratch, the study tests 'repurposed' drugs. These are medicines already approved and safely used for other medical conditions, such as diabetes, high blood pressure, or Parkinson's disease. Because doctors already know a lot about how these medicines work and their safety, researchers can test them much faster to see if they offer any benefit for ALS.

To find out if a drug is working, the research team measures a protein called neurofilament light chain (NFL) in your blood. When motor nerves are damaged, they leak NFL into the bloodstream. If a trial drug lowers NFL levels, this shows it may be helping to protect nerves from further damage. Drugs that show clear promise can then quickly move into larger clinical trials.

Key takeaways

Tests repurposed, existing medicines to see if they can slow down ALS.
Every participant receives an active medication; there are no dummy pills.
Uses a simple blood test (NFL level) to see if drugs protect nerve cells.
Involves taking a medication for up to six months with hospital and remote check-ups.
Helps researchers rapidly choose the most promising treatments for larger trials.

Who may be eligible?

This trial is open to adults aged 18 and older who have a confirmed diagnosis of ALS. To take part, your condition must fall within a specific disease progression range determined by your study doctor. If you are taking riluzole, you must have been on a stable dose for at least one month before starting, or have chosen not to take it.

You cannot join if you are taking part in another drug trial, or if you have other serious illnesses affecting your heart, liver, or kidneys that could make taking the study medicines unsafe. You also cannot join if you have another active condition that alters nerve protein levels in the blood.

Because the study compares different medicines, you must be medically able to take at least two of the drugs being tested. Those who need breathing machines for more than 22 hours a day, or who are pregnant or breastfeeding, are not eligible 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.

I am aged 18 or over and have a confirmed diagnosis of ALS.
I have been on a stable dose of riluzole for at least 30 days, or I do not take it.
I am not currently taking part in another drug trial.
I do not use a breathing machine for more than 22 hours a day.
I am not pregnant or breastfeeding, and I am willing to use effective contraception if required.

Answer every question to see your result.

What does participation involve?

If you join the study, you will take an active study drug for up to six months. There is no dummy pill (placebo), so you and your doctor will know exactly which medication you are receiving. Before you start, the team will check your medical history and take baseline measurements, including blood tests.

During the six months, you will attend hospital visits so the medical team can monitor your health and check for any side effects. To make taking part easier, appointments at week four and week eight can often be done from home by phone or video call. You will also have three fasting blood tests: one at the start, one at week 12, and one at week 24. For these visits, you will be asked not to eat from 8:30 pm the evening before and drink only water until after your morning blood test.

Travel costs for hospital visits can be reimbursed. The study team will stay in regular contact with you, and you can stop taking the medication or leave the study at any point without it affecting your usual NHS care.

Potential risks and benefits

Taking part may not directly improve your own health, but you will be playing a vital role in identifying treatments that could help people with ALS in the future. Because all the study medicines are already used in the NHS for other conditions, their common side effects are well known. Your study team will explain these in detail before you decide to join and will monitor you closely throughout. If you do have side effects, your doctor can lower the dose or stop the medicine. You are always free to leave the trial at any time, for any reason, and your regular NHS medical care will not be affected.

Locations (15)

Royal Hallamshire Hospital
Approximate
Sheffield, England
Oxford Radcliffe Hospital NHS Trust
City only
Oxford, England
The Anne Rowling Regenerative Neurology Clinic
City only
Edinburgh, Scotland
Royal Devon University Healthcare NHS Foundation Trust
City only
Exeter, England
King's College Hospital
Approximate
London, England
Salford Royal Hospital
Unverified
Salford, England
University Hospital of Wales
Unverified
Cardiff, Wales
University College Hospital
Unverified
London, England
Royal Victoria Infirmary
Unverified
Newcastle upon Tyne, England
The Walton Centre NHS Foundation Trust
Unverified
Liverpool, England
St George's Hospital
Unverified
London, England
Western Health and Social Care Trust
Unverified
Londonderry, Northern Ireland

Common questions

Will I receive a dummy pill (placebo)?

No. In this trial, there is no dummy pill. Every participant receives one of the active study medications.

Can I stay on my regular ALS medication, like riluzole?

Yes, provided you have been on a stable dose of riluzole for at least 30 days before joining the study.

How long will I take the study drug?

You will take the assigned study medication for up to six months, with regular check-ups to monitor your progress.

Do I have to travel to the hospital for every visit?

No. While some visits require blood tests at the hospital, your week 4 and week 8 check-ups can usually be done by video or phone.

What happens if I experience unpleasant side effects?

You should tell your study team straight away. They can adjust your dose, pause the medication, or stop it completely if necessary.

How to find out more

Jennifer Petrie

experts-als@sheffield.ac.uk None provided Official trial record

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