Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

A study to evaluate the effectiveness and safety of Usnoflast in adults with Amyotrophic Lateral Sclerosis (ALS)

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

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

This study focuses on Amyotrophic Lateral Sclerosis, also known as ALS, which is a rare disease that affects the nerve cells in the brain and the spinal cord. This condition causes the muscles to weaken over time. The purpose of the study is to evaluate the efficacy and safety of the drug Usnoflast. Participants in the study will receive either Usnoflast in the form of a capsule or a placebo, which is a substance that looks exactly like the study drug but contains no active medicine.

During the course of the study, the way the body processes the medication, known as pharmacokinetics, and the drug's effects on the body, known as pharmacodynamics, will be observed. Researchers will also look at NfL, which is a specific protein found in the blood and cerebrospinal fluid—the clear liquid that surrounds the brain and spinal cord—that can indicate nerve cell damage. The study will monitor how the disease progresses and look at how the treatment affects physical functions and survival over a period of time.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>study participation</b>

    Participation in this study involves receiving either the test medication, called usnoflast, or a placebo, which is a pill that looks, feels, and acts like the medication but contains no active substance.

    The assignment of the medication is randomized, meaning it is decided by chance, and double-blind, meaning neither the participant nor the researchers know which substance is being taken.

  2. Step 2

    <b>medication administration</b>

    The usnoflast or the placebo is taken as a capsule by oral use (swallowing by mouth).

  3. Step 3

    <b>monitoring and assessment</b>

    The study duration for receiving the medication lasts for 36 weeks.

    During this time, disease progression is monitored using the ALSFRS-R total score, which is a scale used to measure how much the disease affects physical functions.

    Lung function is measured through SVC, which refers to the forced vital capacity, a test to see how much air the lungs can hold.

    Quality of life is assessed using the ALSAQ-40 score, which is a questionnaire regarding daily living.

    Levels of a protein called NfL are measured in the serum (the liquid part of the blood) and the CSF (cerebrospinal fluid, which is the fluid surrounding the brain and spinal cord) to track changes.

    Blood plasma and CSF are sampled at baseline (the start), at week 16, and at week 36 to study how the body processes the medication, a process known as pharmacokinetics.

    Safety is monitored by tracking TEAEs (treatment-emergent adverse events, or side effects that happen during the study) and SAEs (serious adverse events, or more severe medical problems) up to week 36.

Who can join the trial?

9 criteria

  • Participants must be men or women who are 18 years of age or older at the time of the initial evaluation.
  • Participants must have a diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS), which is a condition that affects nerve cells in the brain and spinal cord, based on specific medical standards.
  • The first symptoms of the condition must have started within the last 24 months.
  • Participants must have an ALSFRS-R score of 35 or higher, which is a scale used by doctors to measure how much the disease affects a person's daily physical functions.
  • The forced vital capacity (FVC), which is a measurement of how much air your lungs can hold, must be at least 60% of what is expected for a healthy person of your age and size.
  • Participants must be able to swallow capsules.
  • Participants must either not be taking certain medications like riluzole, sodium phenylbutyrate, taurursodiol, or tofersen, or they must have been taking a stable dose (the same amount) of these medicines for at least 4 weeks before the study begins.
  • If taking edaravone, participants must have finished at least one cycle of treatment and must continue taking the same dose throughout the study.
  • Participants must be able to give formal permission to join the study and follow all the required rules and steps of the research.

Who cannot join the trial?

25 criteria

  • You have an unstable psychiatric disease (mental health condition), cognitive impairment (problems with thinking, memory, or reasoning), dementia (a progressive loss of memory and mental abilities), or substance abuse (drug or alcohol misuse) that makes it difficult to give legal permission to join the study.
  • You have had an active herpes zoster (shingles) infection within the last 2 months.
  • You have had any condition that caused suicidal thoughts or behavior within the last 6 months that might make it unsafe for you to participate.
  • You have a history of severe or unstable problems with your heart, lungs, cancer, liver, or kidneys, or any other serious illness besides ALS.
  • You have a known allergy or sensitivity to the study drug (the medication being tested) or any of its excipients (inactive ingredients used to make the pill or liquid).
  • You have taken specific medications that affect how the body processes drugs using certain enzymes (proteins that break down medicine) within 7 days of starting the study.
  • You have used steroids, colchicine (medicine used for gout), or anti-IL-1 inhibitors (medicines that block specific parts of the immune system) within 7 days of starting the study.
  • You are currently using or have recently used an investigational drug (a medicine being tested in other research studies) within the last 4 weeks.
  • You use products like St. John’s Wort (a herbal supplement) or other items that change how the body absorbs, metabolizes (breaks down), or eliminates (gets rid of) medicine.
  • You are currently receiving an elemental diet or parenteral nutrition (nutrients delivered directly into the bloodstream through a tube).
  • You have received a blood transfusion within the last 3 months.
  • You have any other medical condition or laboratory significant value (unusual results from blood or urine tests) that the doctor believes prevents you from participating safely.
  • You have HIV, hepatitis B, hepatitis C, coronary artery disease (heart disease), or an active gastrointestinal condition (stomach or bowel issue) that could stop the medicine from being absorbed properly.
  • You are unable to have a venipuncture (a procedure where a needle is inserted into a vein to draw blood) or cannot tolerate it.
  • You or your family members are employees of the person running the study or the location where the study is being held.
  • Any other condition that the doctor believes might make it unsafe for you to participate or might affect the results of the study.
  • Women who are breastfeeding, pregnant, or planning to become pregnant, or those who are unwilling to use effective contraception (birth control) during the study and for 1 month after. Men who are unwilling to use effective contraception during the study and for 1 month after.
  • You have received a live vaccine within the last 14 days or plan to get one during the study.
  • You have ever received stem cell therapy or gene therapy (treatments that use cells or genetic material) for ALS.
  • Your blood tests show high levels of ALT or AST (liver enzymes), high bilirubin (a yellow substance in the blood), or a low estimated glomerular filtration rate (a measure of how well your kidneys filter blood).
  • If you are taking part in the optional spinal fluid collection, you have conditions that prevent a lumbar puncture (a procedure where a needle is inserted into the lower back to collect fluid), such as scoliosis (curvature of the spine), coagulopathy (blood clotting problems), or an infection at the site.
  • You have a history of epilepsy (seizures) within the last 6 months.
  • You have had surgery in the last 3 months or have a major surgery planned in the next 3 months.
  • You have had a serious illness like pneumonia (lung infection) or septicemia (blood infection) within the last 4 weeks, or an infection that required you to stay in the hospital or receive medicine through an IV.
  • You have a chronic bacterial infection, such as tuberculosis, that the doctor deems unacceptable for participation.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

RecruitingNot yet recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Usnoflast is an experimental medication taken by mouth in capsule form that is being studied to see if it can help improve symptoms and increase survival in adults living with Amyotrophic Lateral Sclerosis (ALS).

What is already known about the treatment

Usnoflast - This medication is taken by mouth in the form of a capsule and is currently being studied in clinical trials to treat Amyotrophic Lateral Sclerosis (ALS). As an experimental orphan drug, it is being investigated for its ability to help manage this rare nervous system disease. While its exact molecular workings are still being researched, it is classified as a specialized therapeutic agent designed to influence the progression of nerve cell damage.

Investigated diseases

Amyotrophic Lateral Sclerosis - This is a progressive disease that affects the nerve cells responsible for controlling voluntary muscle movement. It involves the gradual breakdown and loss of motor neurons in the brain and spinal cord. As these nerve cells decline, the ability to send signals to the muscles is impaired. This leads to a loss of muscle strength and coordination throughout the body. The condition typically advances by affecting various bodily functions over time.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-522580-15-00Protocol codeUSNO.24.002Estimated enrolment244 patientsSponsorZydus Therapeutics Inc.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.