A Study Testing the Safety of LTX-002 Sodium Given into the Spinal Canal in Adults with Amyotrophic Lateral Sclerosis

What is this study about?

This study is looking at Amyotrophic Lateral Sclerosis, a disease that affects nerve cells in the brain and spinal cord, leading to loss of muscle control and weakness. The treatment being tested is called LTX-002, which is an antisense oligonucleotide. This medication works by targeting specific genetic material in cells to potentially slow down or change the disease process. Some participants will receive LTX-002 while others will receive placebo. The medication will be given as an injection directly into the space around the spinal cord, which is called intrathecal administration.

The purpose of this study is to evaluate the safety and tolerability of LTX-002 when given to adults with Amyotrophic Lateral Sclerosis. The study will look at different dose levels of the medication to understand how well it is tolerated by participants. During the study, participants will receive multiple doses of either LTX-002 or placebo through injections into the spinal canal. The study team will monitor participants closely through regular visits and various tests.

Throughout the study, several measurements will be taken to assess safety and how the medication moves through the body. These include checking for any side effects or adverse events, performing blood and urine tests, taking heart tracings using electrocardiograms, measuring vital signs like blood pressure and heart rate, and conducting physical and neurological examinations. The study will also measure the levels of LTX-002 in the cerebrospinal fluid, which is the liquid surrounding the brain and spinal cord, and in the blood to understand how the medication is processed by the body. Participants may need to take the medication over several doses and will be followed for a period of time after the last dose to monitor their health and any effects of the treatment.

1 Initial treatment period preparation

On Day 1, your baseline measurements will be recorded. This includes blood pressure, heart rate, breathing rate, and body temperature.

Blood and urine samples will be collected for laboratory testing.

An electrocardiogram (a test that measures the electrical activity of your heart) will be performed.

A physical examination and neurological examination will be conducted to assess your overall health and nervous system function.

2 First dose administration

You will receive your first dose of either LTX-002 or placebo on Day 1.

The medication will be given as an intrathecal injection, which means it will be injected directly into the space around your spinal cord using a bolus injection technique.

The placebo is a solution that looks identical to the active medication but contains no active substance. It consists of sterile artificial cerebrospinal fluid.

Neither you nor the study staff will know whether you are receiving the active medication or placebo during the trial.

3 Monitoring after first dose

After the first injection, your vital signs will be monitored regularly.

Blood samples will be taken at specific times to measure the levels of LTX-002 in your bloodstream.

Samples of cerebrospinal fluid (the fluid surrounding your brain and spinal cord) will be collected to measure drug levels.

Any side effects or health changes will be recorded and assessed.

4 Second dose administration

You will receive a second intrathecal injection of either LTX-002 or placebo.

The timing between doses will be determined by the study protocol.

The same injection procedure will be used as with the first dose.

5 Monitoring after second dose

Vital signs will continue to be monitored.

Blood and cerebrospinal fluid samples will be collected to track medication levels.

Laboratory tests including blood chemistry and blood cell counts will be performed.

Any side effects will be documented.

6 Third dose administration

You will receive a third and final intrathecal injection of either LTX-002 or placebo.

This completes the series of three doses that are part of this study.

7 Monitoring after third dose

Blood samples will be collected at specific times after the final dose to measure medication levels in your bloodstream.

Cerebrospinal fluid samples will be taken to measure drug levels.

Vital signs, electrocardiograms, and laboratory tests will continue to be performed.

Physical and neurological examinations will be conducted.

8 Follow-up period

You will continue to be monitored until Day 169, which is the final study visit.

During this time, regular assessments of your safety will be performed, including vital signs, laboratory tests, and physical examinations.

Any side effects or changes in your health will continue to be recorded.

If you are taking riluzole, edaravone, or sodium phenylbutyrate with taurursodiol for your condition, you should remain on the same stable dose throughout the entire study period until Day 169.

Who Can Join the Study?

You must be between 18 and 75 years old at the time you agree to join the study.
You must have a diagnosis of ALS, which stands for amyotrophic lateral sclerosis, a disease affecting nerve cells in the brain and spinal cord, confirmed using specific medical criteria called Gold Coast criteria.
Your ALS symptoms must have started less than 36 months, which means less than 3 years, before the initial screening visit.
Your SVC, which stands for slow vital capacity and measures how much air you can breathe out slowly, must be at least 50 percent of the expected normal value for someone of your sex, age, and height when measured while sitting.
If you are taking medications for ALS such as riluzole, edaravone, or the combination of sodium phenylbutyrate and taurursodiol, you must have been taking the same dose for at least 30 days before starting the study and plan to continue that dose throughout the study until day 169.
If you are a woman who can become pregnant and have a male partner, or if you are a man with a female partner who can become pregnant, you must agree to use two effective methods of birth control, with at least one being highly effective, from the first day of the study until 3 months after your last dose of the study medication.
You must be able to understand and sign the informed consent form, which is a document explaining the study, before any study procedures begin. You must be able to read and write in the local language. If you understand and agree but cannot physically sign the form, an impartial witness can sign it for you.
You must be healthy enough to undergo the study procedures and attend all required visits, as determined by the principal investigator, who is the main doctor running the study.
Your blood tests measuring coagulation parameters, which show how well your blood clots, including platelet count, International Normalization Ratio, prothrombin time, and activated partial thromboplastin time, must be within normal ranges at screening.
Your body mass index, which is a measure of body fat based on your height and weight, must be between 18 and 40.

Who Cannot Join the Study?

The study information does not provide specific exclusion criteria, which are reasons why someone cannot join this clinical trial
You should ask the study team directly about which medical conditions, medications, or other factors would prevent you from participating
Common reasons people cannot join clinical trials may include having certain other diseases, taking specific medications, or having medical test results outside certain ranges, but these specific details are not listed in the available study information

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Karolinska University Hospital	Solna	Sweden
Afyaadr Odwavpkqtby Ubkgzzuwbwzqn Cqnllxrwwnze Dndie Skezzv E Dawqh Sasmgyd Dc Tlguqy	Turin	Italy

Trial status

Country	Status	Recruitment Start
Germany	Not yet recruiting	01.01.2026
Italy	Not yet recruiting	01.01.2026
Sweden	Recruiting	01.01.2026
The Netherlands	Recruiting	01.01.2026

Trial locations

Investigated drugs:

LTX-002 SODIUM

LTX-002 is an investigational medication being tested in this study. It is given by intrathecal administration, which means it is injected directly into the space around the spinal cord. This medication is being studied to see if it is safe and well-tolerated in adults with amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells in the brain and spinal cord.

Investigated diseases:

Amyotrophic lateral sclerosis

Amyotrophic Lateral Sclerosis – Amyotrophic Lateral Sclerosis is a disease that affects nerve cells in the brain and spinal cord responsible for controlling voluntary muscle movement. The disease causes these nerve cells to gradually break down and die over time. As the nerve cells deteriorate, muscles become progressively weaker because they no longer receive signals from the brain. People with this condition experience increasing difficulty with walking, speaking, swallowing, and breathing as the disease advances. The muscle weakness typically starts in the hands, feet, or limbs and spreads to other parts of the body. Eventually, the disease affects most voluntary muscles throughout the body, leading to severe disability.

Trial ID:

2025-522039-33-00

Protocol code:

LTX-002-101

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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A Study Testing the Safety of LTX-002 Sodium Given into the Spinal Canal in Adults with Amyotrophic Lateral Sclerosis

What is this study about?

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