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Amyotrophic lateral sclerosis – Trials in Disease

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Ongoing Clinical Trials for Amyotrophic Lateral Sclerosis

There are currently 24 clinical trials underway investigating various treatments for Amyotrophic Lateral Sclerosis (ALS), a progressive neurological disease affecting nerve cells in the brain and spinal cord. These trials are testing different approaches including gene therapies, antibody treatments, repurposed medications, and novel compounds, spanning research centres across Europe and beyond. (Also known as: Motor Neurone Disease, MND, Lou Gehrig’s Disease)

Clinical trial locations

Study of psilocybin therapy for psychological distress in patients with COPD, ALS, multiple sclerosis, or atypical Parkinson’s disorders

This trial is testing psilocybin therapy for people with several serious progressive conditions including ALS who are experiencing depression and psychological distress. The main goal is to see whether medium to high doses of psilocybin can safely reduce depression symptoms compared to very low doses.

Who can join: Adults aged 18 or older with a confirmed diagnosis of ALS and significant depression scores. You must meet specific disease criteria including certain minimum scores on physical function tests and have a life expectancy of at least 6 months. You’ll need to have a caregiver or support person, be able to swallow capsules, and willing to attend all study visits.

Who cannot join: People with personal or family history of severe mental health conditions like schizophrenia, bipolar disorder, or psychosis. Those with recent suicide attempts or current suicidal thoughts with plans, active substance abuse, severe heart or organ problems, pregnancy or breastfeeding, or use of certain medications that could interact with psilocybin.

What the study involves: Participants receive two doses of psilocybin capsules (either 1 mg, 15 mg, or 25 mg) taken by mouth over approximately two months. Your mental health will be assessed before treatment and six weeks after the second dose using depression rating scales. The study will also evaluate your quality of life, ability to cope with end-of-life concerns, and overall mental health. The medication is given in a controlled setting with medical supervision.

Treatment being tested: Psilocybin is a naturally occurring psychedelic compound being studied for treating psychological distress and depression in people with serious medical conditions. It works by binding to serotonin receptors in the brain and is given along with psychological support to help patients process their experiences.

Study on Metabolic Impairment in ALS and TIA Patients Using Hyperpolarized Pyruvate MRI

This study is testing a new type of brain imaging using hyperpolarized pyruvate MRI to understand metabolic changes in the brain of people with ALS. The goal is to detect and characterize how the disease affects brain metabolism.

Who can join: Adults between 18 and 85 years old with a confirmed diagnosis of ALS according to the 2021 Gold Coast criteria. You must show upper motor neuron symptoms and have experienced a recent stroke-like event within the last 10 days with symptoms including weakness on one side of the body lasting at least 10 minutes. Pre-menopausal women must have a negative pregnancy test.

Who cannot join: People without a confirmed ALS diagnosis and those under 18 or over 65 years of age.

What the study involves: You will receive an injection of hyperpolarized pyruvate either through a single injection or intravenous infusion. This substance helps make certain metabolic processes visible during an MRI scan. The scan measures how the injected substance is converted to other substances like bicarbonate, lactate, and alanine in the brain. Your health will be monitored through clinical examinations and interviews throughout the study.

Treatment being tested: Hyperpolarized pyruvate is a special form of a natural body substance used in advanced MRI imaging to visualize brain metabolism. This technique helps doctors understand how ALS affects energy production and use in the brain.

Study on Neural Stem Cell and Sodium Chloride Treatment for Patients with Amyotrophic Lateral Sclerosis

This trial is exploring whether human neural stem cells, special cells that can develop into different types of nerve cells, can help slow down the progression of ALS and improve quality of life. The study compares neural stem cells with a placebo to evaluate safety and effectiveness.

Who can join: Adults aged 18 to 65 with a definite or probable ALS diagnosis according to revised El Escorial criteria. The disease should have started within the last 24 months. You need a lung function test result of at least 70%, an ALSFRS-R score of at least 26, and evidence of fast disease progression. You must be on a stable dose of riluzole for more than 30 days or not taking it at all. A caregiver must be available to help with follow-up visits.

Who cannot join: Details about specific exclusion criteria beyond vulnerable populations are not fully specified in the available data.

What the study involves: You will receive human neural stem cells delivered directly into the brain through an intracerebroventricular injection. The study includes regular monitoring for side effects, changes in neurological function, and disease progression. You’ll undergo health assessments, laboratory tests, and heart monitoring throughout the trial period, which extends for several years to gather comprehensive safety and effectiveness data.

Treatment being tested: Human neural stem cells are being tested for their potential to repair or replace damaged nerve cells in the brain and spinal cord. These cells might help slow the progression of ALS or improve symptoms by developing into healthy nerve tissue.

Study on the Effectiveness and Safety of Masitinib with Riluzole for Patients with Amyotrophic Lateral Sclerosis (ALS)

This study evaluates whether adding masitinib to standard riluzole treatment can help slow down the progression of ALS over 48 weeks. The goal is to see if this combination can improve symptoms and quality of life better than riluzole alone.

Who can join: Adults aged 18 to 80 with either familial or sporadic ALS diagnosed within the last 24 months. You must have been taking a stable dose of riluzole (100 mg per day) for at least 12 weeks before joining. Your disease progression rate should fall within a specific range measured by the ALSFRS-R scale. Women of childbearing age and men must agree to use effective contraception. You need to be able to understand and sign the consent form and follow study procedures.

Who cannot join: Specific exclusion criteria are not detailed in the available data.

What the study involves: You will be randomly assigned to receive either masitinib (100 or 200 mg) or placebo, both taken orally alongside your continued riluzole treatment. The study lasts 48 weeks with regular follow-up visits to monitor your condition through physical examinations, blood tests, and assessments using the ALSFRS-R scale. Changes in muscle strength, lung function, and overall health will be tracked throughout.

Treatment being tested: Masitinib is a tyrosine kinase inhibitor that blocks certain enzymes involved in cell growth and survival. It’s being tested to see if it can slow ALS progression when added to standard riluzole therapy. Riluzole is an approved ALS medication that works by reducing the release of glutamate, a neurotransmitter that can be toxic to nerve cells in high amounts.

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

This study is testing whether acetyl-L-carnitine can slow down the loss of self-sufficiency in people with ALS over 48 weeks. Two different doses (1.5 grams and 3 grams per day) are being compared against placebo to find the most effective amount.

Who can join: Adults 18 years or older with normal thinking abilities and a confirmed ALS diagnosis according to Gold Coast Criteria. Your symptoms must have started no more than 24 months ago. You need to be able to take care of yourself with specific minimum scores for swallowing, eating, and walking functions, and have good breathing ability with at least 80% lung function. You must show documented disease progression and have been taking riluzole for at least 4 weeks before starting the study.

Who cannot join: People without an ALS diagnosis, those outside the specified age range, and members of vulnerable populations who cannot give proper consent.

What the study involves: You will receive either acetyl-L-carnitine oral solution (1.5 or 3 grams daily) or placebo for 48 weeks. Regular assessments will track your ability to perform daily activities using the ALSFRS-R scale, along with breathing function, quality of life, and other health indicators. The study monitors whether you maintain self-sufficiency, defined by specific abilities in swallowing, eating, and walking.

Treatment being tested: Acetyl-L-carnitine (ALCAR) is a dietary supplement with potential neuroprotective properties. It’s believed to support energy production in cells and protect nerve cells from damage, potentially slowing functional disability progression in ALS.

Study on the Effects of NX210c and Glucose in Adults with Amyotrophic Lateral Sclerosis (ALS)

This trial is testing NX210c, a synthetic protein given through intravenous infusions, to evaluate its effectiveness and safety in people with ALS. The study focuses on how the treatment affects certain blood and spinal fluid markers related to disease progression.

Who can join: Adults 18 years or older with a confirmed ALS diagnosis with disease duration of 36 months or less from first symptoms. You need specific TRICALS risk profile scores, adequate breathing function (at least 55% lung capacity), and good veins for blood draws. Body weight must be 110 kg or less. Women must not be pregnant or breastfeeding and must use reliable birth control. If taking riluzole, you must have been on a stable dose for at least 30 days. You need to have sufficient spinal fluid available for testing.

Who cannot join: People with other medical conditions besides ALS, those outside the age range, members of vulnerable populations, pregnant or breastfeeding women, and those unable to provide consent.

What the study involves: You will receive multiple intravenous infusions of NX210c or placebo. The treatment includes glucose solution as part of the infusion process. Regular assessments include blood tests, spinal fluid analysis (lumbar punctures), physical examinations, and evaluations of breathing function and overall health. The study tracks changes in specific protein levels (neurofilament light chain) in blood and spinal fluid as markers of disease activity.

Treatment being tested: NX210c is an investigational medication being studied for its effect on blood markers and blood-brain barrier integrity in ALS. It’s administered through multiple intravenous infusions to assess safety, tolerability, and potential effectiveness.

Study on the Effects of Rituximab for Patients with Amyotrophic Lateral Sclerosis (ALS)

This study evaluates whether adding rituximab to standard riluzole therapy can slow down symptom progression in people with sporadic ALS over 79 weeks. Rituximab targets specific immune system cells called B-cells to see if reducing them can benefit disease progression.

Who can join: Adults 18 years or older diagnosed with sporadic ALS for 24 months or less since symptoms started, without needing permanent breathing support. You must be taking riluzole at a stable dose (50 mg twice daily) for at least 30 days and have lung function at least 75% of expected values. You should be up to date with standard vaccinations except Hepatitis B, which will be checked at screening.

Who cannot join: People without sporadic ALS diagnosis, those outside the age range, members of vulnerable populations, and those unable to give informed consent.

What the study involves: You will receive either rituximab or placebo through intravenous infusions, along with your regular riluzole treatment. You may also receive methylprednisolone and paracetamol to help manage potential side effects. Regular follow-up visits will monitor disease progression using the ALS Functional Rating Scale, lung function tests, body weight changes, and overall survival. The study includes safety monitoring through laboratory tests and neuropsychological assessments.

Treatment being tested: Rituximab is a monoclonal antibody that targets and reduces B-cells in the immune system. The study investigates whether depleting these cells might slow ALS progression by affecting the immune system’s role in the disease process.

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

This large international study is testing whether VHB937 can improve survival without permanent breathing support and maintain physical function in people with ALS. After an initial 40-week comparison with placebo, all participants can continue receiving VHB937 in an open-label extension phase.

Who can join: Adults aged 18 to 85 with either sporadic or familial ALS diagnosed according to revised El Escorial criteria. Your ALS symptoms must have started within the last 24 months. You need a lung capacity of at least 60% of expected values and an ALSFRS-R score of 30 or higher. If taking standard ALS medications like riluzole or edaravone, you must have been on stable doses for at least 30 days. You must be able and willing to travel to the study site.

Who cannot join: Specific detailed exclusion criteria are not provided in the available data.

What the study involves: You will receive VHB937 or placebo through intravenous infusion for 40 weeks in a double-blind phase where neither you nor researchers know which treatment you’re receiving. After this, all participants can receive VHB937 in an open-label extension continuing until Week 100. Regular assessments track physical abilities using the ALSFRS-R scale, muscle strength, breathing capacity, quality of life, and safety through vital signs and laboratory tests.

Treatment being tested: VHB937 is a Phase 2 investigational medication being studied for its potential to improve survival and function in ALS patients by targeting disease pathways that may slow progression.

Study on the Safety and Effects of AMT-162 Gene Therapy for Adults with SOD1 ALS

This gene therapy study is testing AMT-162 in adults who have ALS caused by SOD1 gene mutations. The treatment uses a special viral vector to deliver genetic material that may help reduce the effects of the SOD1 mutation.

Who can join: Adults who previously received AMT-162 in a prior study called EPISOD1 at least 6 months ago. You must be willing and able to provide informed consent and understand the study requirements.

Who cannot join: People without confirmed ALS diagnosis, those outside the specified age range, those who cannot safely receive the treatment due to other health conditions, pregnant or breastfeeding women, recent participants in other clinical trials, those with allergies to similar treatments, and those unable to comply with study procedures.

What the study involves: AMT-162 is administered as a single dose through intrathecal injection directly into the spinal canal. The study monitors safety and tolerability with regular assessments including blood tests, cerebrospinal fluid analysis, and evaluations of lung function and muscle strength. Long-term follow-up measures specific protein levels (neurofilament light chain) in the blood for up to 5 years after treatment.

Treatment being tested: AMT-162 is a gene therapy using an adeno-associated viral vector to deliver genetic material targeting the SOD1 gene mutation. It aims to reduce harmful protein production and potentially slow disease progression in SOD1-related ALS.

Study on the Safety and Efficacy of ARGX-119 for Adults with Amyotrophic Lateral Sclerosis (ALS)

This study evaluates the safety, tolerability, and effectiveness of ARGX-119, a monoclonal antibody targeting a specific protein involved in muscle function. The trial includes a double-blind comparison with placebo followed by an active-treatment extension phase.

Who can join: Adults aged 18 to 80 with an ALS diagnosis according to Gold Coast criteria. You need a TRICALS risk profile score between -6.0 and -2.0 and a lung capacity of at least 60% of expected values. Both males and females are eligible, but certain health conditions or treatments may affect participation.

Who cannot join: People without confirmed ALS diagnosis, those outside the age range, individuals unable to comply with study procedures, and members of vulnerable populations.

What the study involves: You will be randomly assigned to receive either ARGX-119 or placebo through intravenous infusions in a double-blind manner. Regular monitoring includes checking for side effects, conducting laboratory tests, measuring vital signs and ECGs, and evaluating muscle function using electrophysiological scans. The study measures how your body processes the medication and monitors for any immune responses. Follow-up continues until April 2027.

Treatment being tested: ARGX-119 is a humanized monoclonal antibody being investigated as a potential treatment for ALS. It works by targeting specific proteins to potentially slow disease progression and is administered intravenously.

Study of Cromolyn Inhalation Treatment for Patients with Mild to Moderate Amyotrophic Lateral Sclerosis (ALS)

This study is testing inhaled cromolyn (PHENOGENE-1a) as an additional treatment for mild to moderate ALS to see if it can help slow functional decline over 24 weeks when used alongside standard therapy.

Who can join: Adults aged 18 to 75 with confirmed ALS diagnosis within the past 24 months. You need a breathing capacity above 70% of normal, an ALSFRS-R score of 38 or higher, and ability to generate sufficient airflow for inhaler use (at least 100 liters per minute). You must be taking stable doses of riluzole for at least 4 weeks. Women who can become pregnant and men must use effective birth control during and for 3 months after the study.

Who cannot join: People under 18 or over 80, pregnant or breastfeeding women, those with severe allergies to medications, recent participation in other trials, significant liver or kidney disease, inability to take oral medications, severe respiratory problems requiring permanent ventilation, ALS diagnosed more than 5 years ago, mental conditions affecting consent, recent drug or alcohol abuse, and severe heart conditions.

What the study involves: You will use an inhaler to take either PHENOGENE-1a or placebo powder capsules while continuing your regular riluzole medication. Treatment lasts 24 weeks with regular assessments of breathing function, ALS symptoms using the ALSFRS-R scale, and overall health monitoring. The maximum daily dose is 64.8 mg.

Treatment being tested: Cromolyn (PHENOGENE-1a) is an anti-inflammatory medication being studied as an inhaled treatment. It works by stabilizing mast cells and preventing release of inflammatory chemicals. While traditionally used for asthma, it’s being explored for potential neuroprotective properties in ALS.

Summary

The 24 ongoing clinical trials for Amyotrophic Lateral Sclerosis represent diverse research approaches spanning traditional pharmaceuticals, gene therapies, stem cell treatments, and novel compounds. Several notable patterns emerge from this research landscape.

European countries show strong representation in ALS research, with France, Spain, Sweden, and Germany each hosting multiple trials. The Netherlands, Belgium, and Italy also serve as important research centres. This geographic distribution ensures broad access to experimental treatments across Europe while allowing researchers to recruit diverse patient populations.

The trials test varied therapeutic strategies. Several studies explore add-on therapies to riluzole, the standard ALS medication, including masitinib, dazucorilant, and acetyl-L-carnitine. Gene therapy approaches target specific genetic mutations, particularly SOD1-related ALS, with treatments like AMT-162, tofersen, and ION363. Other innovative approaches include monoclonal antibodies (ARGX-119, rituximab, AP-101), repurposed medications (lithium, salbutamol, Triumeq), and combination therapies.

Most trials focus on safety and efficacy in slowing disease progression, typically measured through the ALSFRS-R functional rating scale and survival without permanent breathing support. Study durations commonly range from 24 to 48 weeks, with some gene therapy trials including longer follow-up periods extending to several years.

Eligibility criteria generally target adults with relatively recent diagnosis (typically within 18-24 months of symptom onset) who maintain reasonable breathing function and physical abilities. This approach allows researchers to detect treatment effects before advanced disease stages make assessment more difficult.

The variety of mechanisms being tested—from neuroprotection and inflammation reduction to gene silencing and stem cell replacement—reflects the complexity of ALS and the ongoing search for effective treatments for this challenging condition.

Ongoing Clinical Trials on Amyotrophic lateral sclerosis

  • Study of Cromolyn Inhalation Treatment for Patients with Mild to Moderate Amyotrophic Lateral Sclerosis (ALS)

    Recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Czechia Germany Poland Spain

  • A study to evaluate the effectiveness and safety of pridopidine hydrochloride in patients with amyotrophic lateral sclerosis

    Recruiting

    1 1
    Investigated diseases:
    Belgium France Germany Ireland Italy The Netherlands +3

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

    Recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Germany Italy The Netherlands Sweden

  • Study on Metabolic Impairment in ALS and TIA Patients Using Hyperpolarized Pyruvate MRI

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Denmark

  • Study on the Effects of ILB and Riluzole for Patients with Amyotrophic Lateral Sclerosis (ALS)

    Recruiting

    1 1
    Investigated diseases:
    Norway

  • Study on the Effects of EH-301, N-Acetylcysteine, and Riluzole in Patients with Amyotrophic Lateral Sclerosis (ALS)

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Spain

  • Study on the Effects of Acetylcarnitine Hydrochloride in Patients with Amyotrophic Lateral Sclerosis (ALS)

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on Neural Stem Cell and Sodium Chloride Treatment for Patients with Amyotrophic Lateral Sclerosis

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study on the Effects of Rituximab for Patients with Amyotrophic Lateral Sclerosis (ALS)

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • A study to evaluate the safety of VTx-002 in patients with amyotrophic lateral sclerosis.

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium The Netherlands Spain

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