A Study of Long-Term Safety, Tolerability, and Clinical Outcomes of Intrathecally Administered LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis: A Multicenter, Open-Label, Long-Term Extension of Study J6I-MC-OWAA
Amyotrophic lateral sclerosis (ALS), often called motor neurone disease (MND) in the UK, causes nerve cells to gradually stop working, leading to muscle weakness. This study is testing an experimental medicine named LY4256984 to see if it is safe and well tolerated over a longer period. The trial is an 'extension study' for adults who have already completed an earlier trial with this drug (known as study J6I-MC-OWAA). Because it is 'open-label', everyone who takes part will receive the active study medicine; there is no dummy drug (placebo). The treatment is given as an injection directly into the spinal fluid in the lower back. Doctors will monitor participants closely over time to look for side effects and to see how their physical abilities, breathing, and daily activities progress while receiving the medicine.
At a glance
What is this study about?
Amyotrophic lateral sclerosis (ALS), often referred to in the UK as motor neurone disease (MND), is a condition that affects nerves in the brain and spinal cord. Over time, messages from the brain cannot reach the muscles properly, leading to progressive muscle weakness, stiffness, and difficulty with everyday tasks such as walking, talking, or swallowing. There are currently very few treatments available, which is why scientists are working to develop new options.
This clinical trial focuses on an experimental medicine called LY4256984. It is delivered through an intrathecal injection, which means it is gently injected into the fluid that cushions the spinal cord. Giving the drug this way allows it to reach the nervous system directly, where it can have the best chance of working.
This particular study is a 'long-term extension'. That means it is open only to people who have already taken part in and finished the earlier phase of the trial. Its main purpose is to find out what happens when people continue receiving the drug over a longer period. Doctors want to know whether the treatment remains safe, what side effects might appear over time, and whether it helps slow down the progression of symptoms.
Key takeaways
- Tests the long-term safety of the experimental medicine LY4256984 for ALS (motor neurone disease).
- Open only to adults who have completed the earlier trial (study J6I-MC-OWAA).
- All participants receive the real medicine (no placebo).
- The treatment is given as an injection into the spinal fluid.
- Doctors will monitor side effects and physical function over an extended period.
Who may be eligible?
This study is open to adults aged 18 and older who have sporadic ALS (the most common form of motor neurone disease, which does not run strongly in families).
Because this is an extension study, participants must have already taken part in and completed the previous clinical trial testing this medicine (study J6I-MC-OWAA). You cannot join if you did not complete that earlier trial.
You will also need to be well enough to safely have regular injections into your lower back and to travel to the hospital for your appointments. Your study team will review your health history and test results to ensure that continuing the treatment remains safe for you.
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 18 years of age or older?
- Do you have a diagnosis of sporadic ALS (motor neurone disease)?
- Did you complete the earlier study (J6I-MC-OWAA) using this treatment?
- Are you able to attend regular hospital appointments for check-ups and treatment?
- Are you able to undergo a procedure to receive medication into your spinal fluid?
What does participation involve?
Taking part involves visiting a hospital specialist clinic on a regular schedule. During these visits, a doctor will give you LY4256984 using a lumbar puncture, where a fine needle is placed between the bones of your lower back into the spinal fluid. You will have routine medical checks, such as blood tests, heart checks (ECGs), and assessments of your movement, muscle strength, and breathing. You will also be asked how you are feeling and if you have noticed any new symptoms. The study lasts over an extended period of several months or years, and the team will follow your progress closely throughout.
Potential risks and benefits
Locations (4)
- —UnverifiedNetherlands
- —UnverifiedBelgium
- —UnverifiedGermany
- —UnverifiedSpain
Common questions
What is an extension study?
An extension study allows patients who completed a previous trial to keep receiving the experimental treatment, helping doctors gather longer-term safety information.
Will I receive a placebo (dummy medicine)?
No. This is an 'open-label' study, which means every person who participates will receive the active study drug.
How is the medicine given?
The drug is given by an injection into the lower back (a lumbar puncture) so that it goes straight into the fluid surrounding your spinal cord.
Can I join if I was not in the first trial?
No. This study is only open to people who have already completed the previous study (J6I-MC-OWAA) for this medicine.
Will taking part cost me anything?
The study medicine and all trial-related tests are provided free of charge, and trial sponsors often help cover reasonable travel expenses.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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