RecruitingPHASE1INTERVENTIONAL

A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis

This research study is testing a new drug called LY4256984 for people living with Amyotrophic Lateral Sclerosis (ALS), often known as Motor Neurone Disease. The main goal is to understand how safe the new drug is and what side effects people might experience. Researchers will also be checking how the body processes the medication and how quickly it's removed. Half the participants will receive the new drug, and the other half will receive a dummy treatment (placebo) for comparison. The study medicine is given through a special injection directly into the spine. This is an early-stage study, focusing on safety before looking at how well the drug works.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Eli Lilly and Company

Enrolment target

Start

05 Aug 2025

Estimated completion

01 Sep 2027

ALS (Amyotrophic Lateral Sclerosis)

What is this study about?

This study is about a new medicine called LY4256984 for people who have sporadic 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 muscle weakness over time. This particular medicine is still in its early stages of testing.

The main purpose of this study is to check how safe LY4256984 is and what side effects it might cause. When new medicines are developed, doctors need to first make sure they are safe for people to take. They also want to understand how the body handles the medicine – how much of it gets into the blood and how quickly the body gets rid of it.

To find this out, some people in the study will get the new medicine, while others will get a placebo. A placebo is a dummy treatment that looks just like the real medicine but contains no active drug. This helps researchers compare the effects of the new medicine fairly. The study medicine, or placebo, will be given directly into the fluid around the spinal cord through an injection. This method is chosen to help the medicine reach the areas it needs to target more effectively.

Key takeaways

This study is testing a new medicine (LY4256984) for ALS.
It's a Phase 1 study, mainly focused on safety and side effects.
The medicine is given by an injection into the spine.
Some people will get the active medicine, others a placebo (dummy treatment).
Eligible participants are aged 18-75 with a recent ALS diagnosis.
Strict health conditions apply to ensure patient safety.

Who may be eligible?

To join this study, you need to be an adult between 18 and 75 years old and have been diagnosed with sporadic ALS (Motor Neurone Disease). Your ALS symptoms must have started within the last two years. Your body mass index (BMI), which is a measure of your weight compared to your height, needs to be between 18 and 35.

There are certain reasons why you might not be able to join. For example, if you have other serious health problems that could make it unsafe to take part, such as significant issues with your heart, kidneys, liver, or lungs. You also couldn't join if you have another brain-related disease, severe memory problems, or if your blood tests show certain high levels, or if you have severe kidney problems.

Also, if a heart tracing (ECG) shows an abnormal pattern that the doctors think could put you at risk, or if previous scans of your spine show problems, you might not be able to participate. These rules are to ensure your safety throughout the study.

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 75 years old?
Do you have a diagnosis of sporadic ALS (Motor Neurone Disease)?
Did your ALS symptoms start within the last two years?
Is your BMI (weight-for-height) between 18 and 35?
Do you have good overall health without other serious medical conditions?
Do you have normal kidney and liver function?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be given either the new medicine, LY4256984, or a placebo (a dummy treatment). This will be given as an injection into the fluid around your spine. Throughout the study, you will have regular check-ups, which will include blood tests to see how your body is reacting to the medicine and how much of it is in your system. This helps the doctors understand its effects and how quickly your body gets rid of it. The exact number of visits and the full duration of your participation will be explained in detail by the study team.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit of receiving a new experimental treatment for ALS, which could theoretically help manage your condition, though this is not guaranteed as it's an early-stage study. However, there are also potential risks including side effects from the study drug or the injection procedure itself. The research team will carefully monitor you for any problems. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (12)

UZ Leuven
Verified postcode
Leuven, Belgium· Recruiting
Heritage Medical Research Clinic
Verified postcode
Calgary, Canada· Not yet recruiting
Walter Mackenzie Health Sciences Centre
Verified postcode
Edmonton, Canada· Not yet recruiting
London Health Sciences Centre
Verified postcode
London, Canada· Not yet recruiting
Montreal Neurological Institute and Hospital
Verified postcode
Montreal, Canada· Recruiting
Sunnybrook Research Institute
Verified postcode
Toronto, Canada· Recruiting
Universitätsklinikum Schleswig-Holstein
Verified postcode
Lübeck, Germany· Recruiting
Universitätsmedizin Rostock Sektion für Translationale Neurodegeneration "Albrecht Kossel" Klinik und Poliklinik für Neurologie
Verified postcode
Rostock, Germany· Not yet recruiting
Universitätsklinikum Ulm
Verified postcode
Ulm, Germany· Not yet recruiting
Universitair Medisch Centrum Utrecht
Verified postcode
Utrecht, Netherlands· Recruiting
Hospital Universitario de Bellvitge
Verified postcode
L'Hospitalet de Llobregat, Spain· Recruiting
Hospital Universitari i Politecnic La Fe
Verified postcode
Valencia, Spain· Recruiting

Common questions

What is ALS?

ALS, or Amyotrophic Lateral Sclerosis, is a serious condition that affects nerve cells in the brain and spinal cord, leading to muscle weakness and problems with movement.

What is a placebo?

A placebo is a 'dummy' treatment that looks exactly like the real medicine but doesn't contain any active drug. It helps researchers compare if the new medicine truly has an effect.

How is the study medicine given?

The study medicine is given as an injection directly into the fluid surrounding your spinal cord. This is called an intrathecal injection.

What is the main goal of this study?

The main goal is to find out if the new medicine, LY4256984, is safe and what side effects it might cause in people with ALS.

Can I stop participating in the study if I change my mind?

Yes, you can choose to stop participating in the study at any time, for any reason, and it won't affect your ongoing medical care.

How to find out more

Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or

lillytrials@lilly.com 1-317-615-4559 Official trial record

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