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Study on the Effects of ION363 for Patients with Amyotrophic Lateral Sclerosis with Fused in Sarcoma Mutations

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Amyotrophic Lateral Sclerosis (ALS), specifically in patients who have a genetic change known as Fused in Sarcoma mutations (FUS-ALS). ALS is a disease that affects the nerve cells in the brain and spinal cord, leading to a loss of muscle control. The study is testing a treatment called ION363, which is an injection designed to target these specific genetic changes. The purpose of the study is to evaluate how effective and safe ION363 is for people with FUS-ALS.

Participants in the study will receive either the ION363 treatment or a placebo, which is a substance with no active medication. The treatment is given through a method called intrathecal use, meaning it is injected into the space around the spinal cord. The study will monitor participants over time to see how the treatment affects their ability to function and survive. This includes looking at changes in their physical abilities and overall quality of life.

The study will also measure various biological markers, such as levels of certain proteins in the blood and spinal fluid, to understand the treatment's impact on the disease. The trial is expected to continue for several years, allowing researchers to gather comprehensive data on the long-term effects of ION363 in treating FUS-ALS. Participants will be closely monitored throughout the study to ensure their safety and to collect detailed information on how the treatment affects their condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit and consent

    Upon joining the study, you will have an initial visit at the study center. During this visit, you will be asked to provide written informed consent. This means you agree to participate in the study after understanding all the details and potential risks involved.

    If you are under 18 years old, a parent or guardian must also provide consent and accompany you to all trial visits.

  2. Step 2

    Eligibility confirmation

    The study team will confirm your eligibility to participate. This includes checking for a genetic mutation in the FUS gene, which must be confirmed by a certified laboratory.

    Your ability to meet all study requirements, including travel and procedures, will be assessed.

  3. Step 3

    Baseline assessments

    Before starting the treatment, baseline assessments will be conducted. These assessments include measuring your functional abilities using the ALSFRS-R scale, which evaluates different aspects of your physical function.

    Other assessments may include blood tests and evaluations of your respiratory function.

  4. Step 4

    Treatment administration

    You will receive the study medication, ION363, or a placebo through an injection into the space around your spinal cord. This method is known as intrathecal administration.

    The frequency and duration of the treatment will be determined by the study protocol, and you will be informed about the schedule during your visits.

  5. Step 5

    Regular follow-up visits

    You will have regular follow-up visits at the study center. During these visits, your health and response to the treatment will be monitored.

    Assessments similar to the baseline will be repeated to track any changes in your condition.

  6. Step 6

    End of study assessments

    At the end of the study period, final assessments will be conducted to evaluate the overall effects of the treatment.

    These assessments will help determine the efficacy and safety of ION363 in treating ALS with FUS mutations.

Who can join the trial?

10 criteria

  • Must provide written informed consent, which means you agree to participate in the study after being informed about all aspects of it.
  • Must be at least 10 years old and show signs or symptoms of ALS, a condition affecting nerve cells in the brain and spinal cord.
  • Must have a confirmed genetic mutation in the FUS gene, verified by a certified laboratory.
  • Must have a lung function test result (called SVC) that is at least 50% of the expected value for your age, sex, and height. If it's less than 50%, you must be between 10 and 30 years old and have had ALS symptoms for less than 12 months.
  • Must be able and willing to meet all study requirements, including traveling to the study center and attending all visits.
  • If taking certain medications for ALS, like edaravone or riluzole, you must have been on a stable dose for at least 28 days before starting the study and be willing to continue that dose unless advised otherwise by the study doctor.
  • Females must be non-pregnant and non-breastfeeding. They must either be surgically sterile, post-menopausal, abstinent, or agree to use a highly effective birth control method during the study and for 40 weeks after the last dose of the study drug.
  • Males must be abstinent, surgically sterile, or agree to use a highly effective birth control method if engaging in sexual relations with a woman who can become pregnant, during the study and for 40 weeks after the last dose of the study drug.
  • Must have stable medications and nutritional support for at least 1 month before starting the study, unless the study doctor decides otherwise.
  • Must have a caregiver or informant who can provide accurate information about your abilities throughout the study. If you are under 18, you must have a reliable adult who can accompany you to all study visits.

Who cannot join the trial?

8 criteria

  • Patients with any other serious health condition that might interfere with the study.
  • Patients who are unable to follow the study procedures or instructions.
  • Patients who have participated in another clinical trial recently.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of allergic reactions to the study medication.
  • Patients who have a significant mental health condition that is not well controlled.
  • Patients who have a history of substance abuse that might interfere with the study.
  • Patients who are unable to provide informed consent.
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Investigated drugs

ION363 is a medication being studied for its potential to help patients with a specific type of Amyotrophic Lateral Sclerosis (ALS) that is caused by mutations in a gene called Fused in Sarcoma (FUS). This medication is given directly into the spinal fluid, which is a method known as intrathecal administration. The goal of using ION363 is to see if it can improve how well patients function in their daily lives and if it can help them live longer. Researchers are also looking at how the body processes the medication and how it affects the disease. This study is important because it could lead to a new treatment option for people with this rare form of ALS.

What is already known about the treatment

ION363 – ION363 is administered through an injection into the spinal canal, a method known as intrathecal administration. It is currently being studied in clinical trials for its potential use in treating Amyotrophic Lateral Sclerosis (ALS) in patients with specific genetic mutations known as Fused in Sarcoma (FUS) mutations. The main goal of this medication is to improve clinical functioning and survival in these patients. At the molecular level, ION363 works by targeting and modifying the genetic material involved in the disease process, aiming to reduce the harmful effects of the FUS mutations. It is classified as an investigational drug, meaning it is still under study and not yet approved for general medical use.

Investigated diseases

Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutations – This is a neurodegenerative disease characterized by the progressive loss of motor neurons, which are nerve cells responsible for controlling voluntary muscles. The disease leads to muscle weakness and atrophy, affecting the ability to move, speak, swallow, and eventually breathe. The progression involves a gradual decline in muscle function, starting with subtle symptoms like muscle twitching and stiffness. Over time, individuals experience increasing difficulty with motor tasks, such as walking and using their hands. The disease is associated with mutations in the Fused in Sarcoma (FUS) gene, which plays a role in RNA processing and transport. As the condition advances, it significantly impacts daily activities and quality of life.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2024-512163-31-00Protocol codeION363-CS1Estimated enrolment95 patientsSponsorIonis Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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