RecruitingPHASE1, PHASE2INTERVENTIONAL

FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS

The FUNCtion ALS trial is looking into a new experimental medicine, TRCN-1023, for adults living with Amyotrophic Lateral Sclerosis (ALS). ALS is a serious condition that affects nerve cells. This study will check if TRCN-1023 is safe and how well people tolerate it. The medicine is given as a single injection into the fluid surrounding the spinal cord. Researchers also want to understand how the body handles the medicine and if there are any early signs that it might help with ALS symptoms over 24 weeks. This is an early-stage study to find the best dose and prepare for larger trials if it looks promising.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Trace Neuroscience, Inc.

Enrolment target

Start

20 Aug 2026

Estimated completion

01 Sep 2027

Amyotrophic Lateral Sclerosis

What is this study about?

This study, called FUNCtion ALS, is testing a new medicine known as TRCN-1023. It’s designed for adults who have Amyotrophic Lateral Sclerosis, more commonly known as ALS or Motor Neurone Disease. ALS is a condition that affects the nerve cells in the brain and spinal cord, leading to muscle weakness and problems with movement, speech, and breathing.

The experimental medicine, TRCN-1023, works by trying to fix a problem with a specific protein called UNC13A, which often doesn't work correctly in people with ALS. The idea is that by helping this protein, the medicine might be able to slow down the disease. It's given as a single injection into the fluid around the spine, and it's hoped that this one injection could last for as long as 24 weeks.

This is an early-stage study (called Phase 1/2), which means the main goals are to check the medicine's safety and how well people tolerate it. Researchers will also learn how the body processes the drug and look for any early indications that it could help improve physical function and quality of life for people with ALS. If successful, this information will help them plan bigger studies in the future.

Key takeaways

This is an early-stage study (Phase 1/2) for a new ALS medicine.
The medicine (TRCN-1023) aims to correct a protein problem in ALS.
It's given as a single injection near the spine.
The main goals are to check safety, how well people tolerate it, and early signs of benefit.
Participation involves regular visits, medical tests, and potentially at-home assessments.
You can withdraw from the study at any time.

Who may be eligible?

To be considered for this study, you would need to be an adult between 18 and 75 years old with a diagnosis of ALS. Your ALS symptoms should have started within the last two years. You'd also need to be able to complete breathing tests, showing your breathing capacity is at least 60% of what's expected for your age and size, and be on a steady dose of any approved ALS medication for at least four weeks before joining.

There are some reasons why you might not be able to join. For example, if you have certain genetic types of ALS (like those linked to SOD1 or FUS gene changes), or if you need breathing support for more than 22 hours a day. You also can't take part if you have other medical conditions that might interfere with the study, such as significant liver or kidney problems, or if you have medical devices like a pacemaker that prevent you from having an MRI scan. If you've recently been part of another trial or received similar experimental treatments, you wouldn't be eligible.

The study team will check these and other details carefully to make sure it's safe and appropriate for you to participate.

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 an adult aged 18 to 75 years?
Do you have a diagnosis of ALS (Motor Neurone Disease)?
Did your ALS symptoms start within the last two years?
Are you able to perform breathing tests with a good capacity (at least 60% of expected)?
Are you currently on a stable dose of approved ALS medication?
Have you *not* had certain genetic types of ALS (SOD1 or FUS mutations)?

Answer every question to see your result.

What does participation involve?

If you join the study, you would receive TRCN-1023 or a placebo (a dummy treatment) as a single injection into the fluid around your spinal cord. This is called an intrathecal injection. Over the 24-week study period, you would have regular visits to the study site for various assessments. These include blood tests, breathing tests, brain scans (MRI), and potentially more lumbar punctures to check drug levels. You might also be asked to use wearable sensors and complete speech assessments at home. The study team will explain all visits and tests in detail, and you'll have ongoing support throughout.

Potential risks and benefits

Taking part in a clinical trial may offer the chance to receive a new, experimental treatment that isn't yet available. However, there's no guarantee the treatment will help you, and it might not work better than existing options. There are also potential risks involved, including side effects from the experimental medicine or discomfort from procedures like injections, blood tests, or MRI scans. The study team will explain all known risks. Your safety is a top priority, and you have the right to withdraw from the study at any time, for any reason, without affecting your regular medical care.

Locations (6)

Universitätsklinikum Schleswig-Holstein
Verified postcode
Lübeck, Germany· Recruiting
TUM Klinikum Rechts der Isar
Verified postcode
Munich, Germany· Recruiting
University Medical Center Rostock
Verified postcode
Rostock, Germany· Recruiting
University Medical Center Utrecht
Verified postcode
Utrecht, Netherlands· Recruiting
University College London
Verified postcode
London, United Kingdom· Recruiting
University of Sheffield
Verified postcode
Sheffield, United Kingdom· Recruiting

Common questions

What is ALS?

ALS stands for Amyotrophic Lateral Sclerosis, also known as Motor Neurone Disease. It's a condition that affects nerve cells, leading to muscle weakness and difficulties with movement, speech, and breathing.

What is TRCN-1023?

TRCN-1023 is an experimental medicine being tested in this study. It's designed to help fix a problem with a protein in the body that's often disrupted in people with ALS.

How is the medicine given?

The medicine is given as a single injection into the fluid that surrounds your spinal cord, which is called an intrathecal injection.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks like the real medicine but contains no active drug. It helps researchers compare the effects of the new medicine accurately.

How long will the study last for each person?

Each person in the study will be followed for 24 weeks after receiving the injection.

How to find out more

Trace Neuroscience Clinical Trials

clinicaltrials@traceneuro.com +1 415-390-9509 Official trial record

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