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Study on the Effects of VHB937 in Patients with Amyotrophic Lateral Sclerosis (ALS)

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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 the effects of a new treatment called VHB937 for people with Amyotrophic Lateral Sclerosis (ALS). ALS is a progressive disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and loss of physical function. The study aims to understand how well VHB937 works compared to a placebo in improving survival without the need for permanent assisted ventilation and maintaining function in individuals with ALS.

Participants in the study will receive either VHB937 or a placebo through an intravenous infusion, which means the treatment is given directly into a vein. The study will last for 40 weeks, during which participants will be monitored for changes in their condition. After this period, there will be an open-label extension, where all participants may receive VHB937, allowing researchers to gather more information about the treatment's long-term effects.

The main goal of the study is to compare the effectiveness of VHB937 against a placebo in terms of survival without permanent assisted ventilation and overall function. Researchers will also look at other factors, such as changes in muscle strength, breathing capacity, and quality of life. Safety and any side effects will be closely monitored throughout the study to ensure the well-being of all participants.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and initial treatment

    Upon joining the study, participants are randomly assigned to receive either the experimental drug VHB937 or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.

    The treatment is administered as a concentrate for solution, given through an intravenous infusion. This involves receiving the medication directly into a vein.

  2. Step 2

    Double-blind phase

    The double-blind phase lasts for 40 weeks. During this time, participants continue to receive either VHB937 or the placebo.

    Participants are monitored for changes in their condition, including survival without the need for permanent assisted ventilation and changes in functional ability.

  3. Step 3

    Open-label extension

    After completing the double-blind phase, participants enter an open-label extension phase. In this phase, all participants receive VHB937, regardless of their initial group.

    This phase continues until Week 100 or until the study ends, allowing further observation of the drug's effects over a longer period.

  4. Step 4

    Monitoring and assessments

    Throughout the study, participants undergo regular assessments to monitor their health and the effects of the treatment. These include evaluations of functional ability, quality of life, and vital signs.

    Safety is closely monitored, with attention to any adverse events or changes in laboratory data.

  5. Step 5

    End of study

    The study is estimated to conclude by April 2029. At this point, all data collected will be analyzed to determine the efficacy and safety of VHB937 in treating amyotrophic lateral sclerosis (ALS).

Who can join the trial?

8 criteria

  • You must sign a document called an informed consent before joining the study. This means you agree to participate and understand what the study involves.
  • You need to have a type of condition called Amyotrophic Lateral Sclerosis (ALS), which can be either sporadic (occurring randomly) or familial (running in families). The diagnosis should be made using specific guidelines known as the revised El Escorial criteria.
  • You must be at least 18 years old.
  • Your ALS symptoms, like muscle weakness, should have started within the last 24 months.
  • Your Slow Vital Capacity (SVC), which measures how much air you can exhale slowly, should be at least 60% of what is expected for your age, height, and sex.
  • Your ALSFRS-R total score, which is a way to measure your physical abilities, should be 30 or higher.
  • You can be either treated or untreated with standard ALS therapies. If you are treated, you should be on a stable dose of medications like riluzole or edaravone for at least 30 days, or have completed at least one cycle of edaravone given through an IV.
  • You must be able and willing to travel to the study site.

Who cannot join the trial?

10 criteria

  • Patients who do not have Amyotrophic Lateral Sclerosis (ALS) cannot participate. ALS is a disease that affects the nerve cells in the brain and spinal cord, leading to muscle weakness.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
  • Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
  • Patients who have other medical conditions that might interfere with the study or its results cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are participating in another clinical trial at the same time cannot participate.
  • Patients who have a history of allergic reactions to the study medication or similar medications cannot participate.
  • Patients who have used certain medications recently that might interfere with the study cannot participate.
  • Patients who have a history of drug or alcohol abuse cannot participate.
  • Patients who have a mental health condition that might interfere with their ability to participate in the study cannot participate.
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Investigated drugs

VHB937 is a medication being studied for its potential to help people with Amyotrophic Lateral Sclerosis (ALS). ALS is a condition that affects the nerve cells in the brain and spinal cord, leading to loss of muscle control. The trial is investigating whether VHB937 can improve survival without the need for permanent assisted ventilation and maintain physical function in patients.

What is already known about the treatment

VHB937 – This medication is administered orally and is currently being studied in clinical trials for its potential use in treating Amyotrophic Lateral Sclerosis (ALS). It is in the phase 2 stage of clinical trials, where its effectiveness is being compared to a placebo. The main therapeutic indication for VHB937 is to improve survival and function in patients with ALS, specifically focusing on reducing the need for permanent assisted ventilation. The mechanism of action at the molecular level involves targeting pathways that may help slow the progression of ALS. VHB937 is classified pharmacologically as a potential neuroprotective agent.

Investigated diseases

Amyotrophic Lateral Sclerosis (ALS) – ALS is a progressive neurological disease that affects nerve cells in the brain and spinal cord. It leads to the gradual degeneration and death of motor neurons, which are responsible for controlling voluntary muscles. As these neurons deteriorate, individuals experience muscle weakness and atrophy, often starting in the limbs. Over time, the weakness spreads to other parts of the body, affecting speech, swallowing, and breathing. The progression of ALS varies among individuals, but it typically leads to increasing disability. The exact cause of ALS is not fully understood, and it is considered a rare disease.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-512536-29-00Protocol codeCVHB937B12201Estimated enrolment225 patientsSponsorNovartis Pharma AG

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