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Safety Study of NB-4746 with Riluzole and Placebo in Adults 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

Amyotrophic Lateral Sclerosis is a disease that slowly weakens the muscles used for walking, speaking, swallowing and breathing. As the nerves that control these muscles become damaged, people may notice increasing difficulty with everyday activities and may need help with daily tasks.

The trial is testing an oral capsule called NB-4746 and also includes the already approved medicine Riluzole. Some participants will receive a dummy pill, known as a placebo, so that researchers can compare how the real medicines work. The main goal of the study is to find out whether the new drug is safe for people with the disease, meaning it does not cause unacceptable side effects.

People who join the study will take the assigned capsule each day and will visit the clinic several times for simple check‑ups. During these visits doctors will look at basic health information such as blood pressure and heart rate, draw small amounts of blood for laboratory testing, and perform a quick heart rhythm test. The early part of the study lasts about four weeks, and later phases continue for a longer period to keep monitoring safety.

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

    Baseline assessment

    After joining the study, you will undergo a series of baseline assessments. these include a review of your medical history, physical examination, measurement of vital signs (such as blood pressure and heart rate), laboratory blood tests, and a 12‑lead ecg (electrocardiogram) to record the electrical activity of the heart.

    The purpose of these assessments is to establish your health status before any medication is taken.

  2. Step 2

    Initiation of treatment (phase ib)

    You will begin taking nb‑4746 capsules by mouth. the exact dose, frequency, and duration are determined by the study protocol and will be explained by the study staff.

    The phase ib portion of the trial lasts for 28 days, during which you will continue the assigned medication daily.

  3. Step 3

    Daily medication administration

    Each day you will swallow the prescribed capsule of nb‑4746 with water. if you are assigned to the placebo group, you will receive a matching capsule that does not contain active drug.

    The medication is taken orally, and you should follow the timing instructions provided (for example, with or without food).

  4. Step 4

    Regular safety monitoring

    Throughout the 28‑day period you will attend scheduled clinic visits. at each visit the study team will check for any treatment‑emergent adverse events (new or worsening symptoms), record vital signs, repeat laboratory tests, and perform a 12‑lead ecg.

    These visits may occur weekly or as otherwise specified by the protocol.

  5. Step 5

    End‑of‑phase ib evaluation

    At the conclusion of the 28‑day treatment period a final safety assessment is performed. this includes a summary of any adverse events, a review of vital signs, laboratory results, and the 12‑lead ecg.

    The data are used to assess the safety profile of nb‑4746 in participants with amyotrophic lateral sclerosis.

  6. Step 6

    Continuation into phase 2 (open label extension)

    Participants may continue receiving nb‑4746 in a phase 2 or open label extension phase. the same oral capsule is taken for a longer period, with ongoing safety monitoring similar to the phase ib visits.

    During this phase the focus remains on tracking adverse events, vital signs, laboratory tests, and 12‑lead ecg results.

Who can join the trial?

10 criteria

  • Have a diagnosis of ALS based on the Gold Coast Criteria (a set of guidelines doctors use to confirm ALS).
  • First ALS symptom started 48 months or less before the first study day for Phase 1b, or 24 months or less before the first study day for Phase 2 (symptom onset means the first sign of weakness in the arms, legs, face, or trunk).
  • Be able to sign a consent form and agree to follow the study rules.
  • Be 18 to 80 years old when signing the consent.
  • Score at least 2 on the swallowing section of the ALSFRS‑R (a questionnaire that measures how well you can swallow).
  • Have a slow vital capacity (SVC) of at least 60% of the predicted value, meaning your lung function is at least 60% of what is expected for someone of your age, height, and sex.
  • If you take the medication riluzole, you must have been on the same dose for at least 60 days before the screening blood test.
  • If you take the medication edaravone, you must have completed at least one treatment cycle before the screening blood test.
  • All screening laboratory test results must be within normal ranges.
  • Agree to use birth‑control methods as required by the study for the whole study period.

Who cannot join the trial?

9 criteria

  • Having a tracheostomy (a tube placed in the neck to help breathing) or needing a breathing machine for more than 22 hours each day for at least a week.
  • Being unable to swallow or take medicines by mouth when screened.
  • Being in another study of a new drug or having taken a new drug within the past 30 days or for a time longer than five drug half‑lives (the time it takes for half of the drug to leave the body).
  • Having a known allergy or reaction to the study drug NB-4746 or any of its ingredients.
  • Having other serious health problems that are not ALS, such as heart (cardiovascular), lung (respiratory), liver (hepatic), kidney (renal), stomach or intestine (gastrointestinal), hormone (endocrinological), blood (hematological), or nervous system (neurological) disorders that could change how the drug is absorbed, processed, or removed, increase risk, or make the study results unclear.
  • Any other reason the doctor believes could affect the study’s conduct, results, or your safety.
  • Being pregnant (a positive pregnancy test) at screening or on the first day of the study, or breastfeeding on the first day.
  • Currently taking or planning to take the ALS drug tofersen.
  • Having an International Normalized Ratio (INR) higher than 1.4 (a blood test that measures clotting), a platelet count lower than 50,000 per microliter (few blood cells that help clot), or using blood‑thinning medicines such as warfarin, heparin, or direct oral anticoagulants.
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Investigated drugs

  • NB-4746

    is an experimental oral capsule being tested for the first time in people with amyotrophic lateral sclerosis (ALS). In this study, participants take NB-4746 to see if it is safe to use over several weeks and months. Researchers will watch for any side effects and check how the drug behaves in the body. The goal is to learn whether NB-4746 could be a useful new treatment for ALS.

  • Riluzole

    is an approved oral medication that is already used to treat ALS. In this trial, participants continue taking riluzole while they also receive NB-4746. The purpose of keeping riluzole in the study is to see how the new drug works together with the standard treatment and to ensure participants receive the usual care for their disease.

What is already known about the treatment

  • NB-4746

    This investigational drug is taken as an oral capsule that is swallowed with water. It is currently in early clinical testing and has not yet been approved for any medical use. The study is looking at its safety for treating amyotrophic lateral sclerosis (ALS). NB-4746 is thought to work by protecting nerve cells from damage, and it is classified as an experimental neuroprotective agent.

  • Riluzole

    This medication is also given as an oral capsule that patients swallow. It is an approved drug that has been used for many years to treat ALS. Riluzole helps slow the disease by reducing excess glutamate, a chemical that can harm nerve cells, and it is classified as a glutamate release inhibitor (a type of neuroprotective agent).

Investigated diseases

Amyotrophic Lateral Sclerosis - A disease that damages the nerve cells responsible for controlling voluntary muscles. It begins with subtle muscle weakness, often in the hands or feet, and may cause clumsiness. As the nerves continue to deteriorate, everyday tasks become harder and muscle stiffness may appear. Weakness spreads to other parts of the body, leading to difficulty walking, speaking, or swallowing. Over time, more muscles are affected, and the ability to move independently declines.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase ITrial ID2025-523939-18-00Protocol codeNB-4746-201Estimated enrolment80 patients

sourced from the EU Clinical Trials Register and site verification

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