RecruitingPHASE3INTERVENTIONAL

Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS

There are also some health checks to make sure the study is a good fit for you. For example, your breathing capacity needs to be at a certain level. You will also need to be able to swallow the capsules on day 1. After that, they can be administered via a feeding tube if required.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Prilenia

Enrolment target

500

Start

01 Feb 2026

Estimated completion

01 Mar 2029

Amyotrophic Lateral Sclerosis

What is this study about?

This study is looking into a potential new treatment for Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. ALS is a condition that affects nerve cells in the brain and spinal cord, leading to a loss of muscle control. Currently, there are treatments that can help manage symptoms, but more research is needed to find ways to slow down the disease.

The drug being tested is called pridopidine. Researchers want to know if taking pridopidine can help slow down the changes that happen in ALS. They also want to make sure it's safe for people to use. This kind of study is really important because it helps us understand if new medicines can make a real difference in people's lives.

To find out if pridopidine works, some participants will receive the actual drug, while others will receive a 'placebo'. A placebo looks exactly like the drug but doesn't contain any active medicine. This helps researchers compare the effects of pridopidine fairly. Neither you nor your study doctor will know whether you're taking the actual drug or the placebo during the first part of the study. This helps ensure the results are unbiased.

Key takeaways

Tests a new drug, pridopidine, for ALS.
Aims to see if pridopidine can slow ALS progression and is safe.
Involves taking pridopidine or a dummy pill daily for 48 weeks, then pridopidine for another 48 weeks.
Requires regular clinic or phone check-ups for about two years.
Open to adults with recent ALS diagnosis, aged 18-80.
You can withdraw from the study at any time.

Who may be eligible?

This study is for adults aged between 18 and 80 years old who have been diagnosed with 'definite' or 'probable' ALS. You would typically have started experiencing ALS symptoms within the last 18 months before joining the study.

There are also some health checks to make sure the study is a good fit for you. For example, your breathing capacity needs to be at a certain level. You also need to be able to swallow capsules, as this is how the study medication is taken.

However, some health conditions might mean this study isn't right for you. This includes if you need a breathing tube or constant breathing support. Also, serious heart problems, unstable mental health conditions, or certain genetic mutations linked to ALS might mean you can't join. If you're pregnant or taking certain other medications, you also wouldn't be able 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.

Are you between 18 and 80 years old?
Have you been diagnosed with definite or probable ALS?
Did your ALS symptoms start within the last 18 months?
Can you swallow capsules easily?
Do you currently use a breathing tube or need constant breathing support? (If yes, you likely can't join)
Do you have a severe heart condition, unstable mental health issues, or are pregnant? (If yes, you likely can't join)

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be in it for about 102 weeks (around two years). During the first 48 weeks, you will take either the study drug, pridopidine, or a dummy pill (placebo) twice a day by mouth. You won't know which one you are taking. After these 48 weeks, all participants will then take pridopidine for another 48 weeks.

You will have regular check-ups with the study team. These check-ups will usually happen every 1 to 3 months and will involve visits to the clinic. Sometimes, you might also have virtual visits over the phone. These appointments are important for the medical team to monitor your health, see how you're responding to the treatment, and make sure you're doing okay.

Potential risks and benefits

Participating in a clinical trial like this could potentially offer benefits, such as receiving a new investigational drug that might help manage your ALS, or closely monitoring your health by a team of specialists. However, there are also potential risks; for example, pridopidine might cause side effects, or it might not work for you. It's important to remember that you can stop taking part in the study at any time, for any reason, without it affecting your usual medical care.

Locations (35)

California Pacific Medical Center
Verified postcode
San Francisco, United States· Recruiting
Mayo Clinic
Verified postcode
Jacksonville, United States· Recruiting
University of South Florida
Verified postcode
Tampa, United States· Recruiting
Emory University
Verified postcode
Atlanta, United States· Recruiting
Northwestern University
Verified postcode
Chicago, United States· Recruiting
University of Kansas
Verified postcode
Fairway, United States· Recruiting
Sean M. Healey & AMG Center for ALS
Verified postcode
Boston, United States· Recruiting
Washington University
Verified postcode
St Louis, United States· Recruiting
Somnos Clinical Research
Verified postcode
Lincoln, United States· Recruiting
Eleanor and Lou Gehrig ALS Center at Columbia University
Verified postcode
New York, United States· Recruiting
Neuroscience Department at LKSM at Temple University
Verified postcode
Philadelphia, United States· Recruiting
Texas Neurology
Verified postcode
Dallas, United States· Recruiting

Common questions

What is ALS?

ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease. It's a condition that affects the nerve cells controlling your muscles.

What is a placebo?

A placebo is a substance that looks exactly like the study drug but doesn't contain any active medicine. It helps researchers fairly compare the effects of the actual drug.

How long will I be in the study?

The study will last for about 102 weeks, which is roughly two years, including initial checks and follow-up.

Will I know if I'm taking the real drug?

For the first 48 weeks, neither you nor your doctors will know if you're taking pridopidine or the placebo. After that, everyone will receive pridopidine.

Can I continue my current ALS medications?

Yes, you can usually continue your standard ALS treatments like riluzole, edaravone, or Nuedexta, as long as you've been on a stable dose for at least 4 weeks before starting the study.

How to find out more

Ferrer Medinfo

medinfo@ferrer.com +34 936003739 Official trial record

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