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VORMATRIGINE

Clinical trials are investigating VORMATRIGINE in adults with epilepsy, especially people with focal seizures and primary generalized tonic-clonic seizures. These studies are looking at safety, tolerability, and how well the treatment may reduce seizure frequency. Both trials are Phase 3 studies in adult patients.

Table of Contents

Clinical trials overview

Two Phase 3 trials are investigating VORMATRIGINE in adults with epilepsy.[1][2] Both studies are interventional, which means participants receive a study treatment and the researchers measure the results.[1][2]

The trials are authorised and together focus on adults with focal seizures, focal epilepsy, and primary generalized tonic-clonic seizures.[1][2] The studies are designed to look at both how well the treatment may work and how safe it is in these groups.[1][2]

Trial 1: Open-label extension in adults with epilepsy

The first study is an open-label extension clinical trial in adult patients with epilepsy.[1] It includes adults with focal onset seizures or primary generalized tonic-clonic seizures.[1]

This trial plans to enroll 196 people and is currently authorised.[1] Its brief summary says the goal is to assess the safety and tolerability of VORMATRIGINE in these adults.[1]

The main outcomes include the incidence and severity of treatment-emergent adverse events (TEAEs), which are health problems that appear or get worse after treatment starts.[1] The study also measures discontinuation of study drug because of TEAEs, changes in vital signs, laboratory results, electrocardiogram parameters, and suicidality using the Columbia Suicide Severity Rating Scale (C-SSRS).[1]

Trial 2: Double-blind randomized study in adults with focal seizures

The second study is a double-blind, randomized Phase 3 trial in adults with focal epilepsy.[2] In a double-blind study, neither the participant nor the researcher knows who gets the study drug or placebo during the trial.[2]

This trial plans to enroll 300 adults and is also authorised.[2] The brief summary says it is testing the efficacy and safety of VORMATRIGINE compared with placebo in adults who are currently taking 1 to 3 anti-seizure medicines (ASMs).[2]

The primary outcome is the 50% responder measure, meaning whether a person has at least a 50% reduction in focal seizure frequency from the observation period to the treatment period.[2] This is a common way to measure seizure improvement in epilepsy studies.[2]

Key endpoints and what they mean

Clinical trials use endpoints to decide what result they want to measure.[1][2] In these studies, the endpoints are focused on seizure control, safety, and overall tolerability.[1][2]

  • Safety: whether the treatment causes health problems that matter during the trial.[1]
  • Tolerability: how well participants are able to stay on the treatment without problems that lead to stopping it.[1]
  • Seizure frequency: how often seizures happen over time.[2]
  • Vital signs: basic body measurements such as blood pressure and pulse.[1]
  • Laboratory results: blood or other test results checked during the study.[1]
  • ECG parameters: measurements from a heart tracing test.[1]

Who may participate

These trials are for adults with epilepsy, not children.[1][2] One study includes adults with focal onset seizures or primary generalized tonic-clonic seizures, while the other includes adults with focal epilepsy who are taking 1 to 3 ASMs.[1][2]

The source data do not give full details about all inclusion or exclusion rules, so the exact entry criteria are not fully listed here.[1][2]

Trial design terms explained

Open-label extension means a follow-up study where the treatment is known to everyone in the study.[1] This type of study often helps collect longer-term safety information.[1]

Randomized means participants are assigned by chance to a study group.[2] Placebo means an inactive treatment used for comparison, so researchers can see whether the study drug works better than no active treatment.[2]

Interventional means the study actively gives a treatment and measures what happens next.[1][2] Phase 3 means the research is in a later stage and usually includes more participants than early studies.[1][2]

Trial IDPhaseCondition studiedStatusEnrollment
2025-521640-38-00Phase 3Focal onset seizures or primary generalized tonic-clonic seizuresAuthorised196
2025-524038-24-00Phase 3Focal epilepsyAuthorised300

FAQ

  • What are the VORMATRIGINE trials studying? The trials are studying safety, tolerability, and seizure control in adults with epilepsy. One trial also compares VORMATRIGINE with placebo to see how well it reduces focal seizure frequency.
  • Who can take part in these trials? Adults with epilepsy may be eligible. One study includes adults with focal onset seizures or primary generalized tonic-clonic seizures, and the other includes adults with focal epilepsy who are taking 1 to 3 anti-seizure medicines.
  • What phase are the VORMATRIGINE trials? Both trials are Phase 3 studies. Phase 3 trials are larger studies that help show how well a treatment works and how safe it is in a target patient group.
  • What outcomes are being measured? One trial measures treatment-emergent adverse events, vital signs, lab results, ECG changes, and suicidality. The other trial measures the 50% responder rate, which means the number of people who have at least a 50% drop in focal seizure frequency.
  • How many people are planned for these studies? The open-label extension trial plans to enroll 196 people, and the double-blind randomized trial plans to enroll 300 people. Together, they study adult epilepsy in different ways.

Ongoing Clinical Trials on VORMATRIGINE

  • Study of PRAX-628 to evaluate safety and effectiveness in adults with epilepsy who have focal seizures or generalized tonic-clonic seizures

    Recruiting

    3 1 1
    Investigated drugs:
    Germany Italy Poland Spain

  • A study to evaluate the effectiveness and safety of vormatrigine in adults with focal epilepsy who are taking one to three anti-seizure medications.

    Not yet recruiting

    3 1
    Investigated drugs:
    Czechia Germany Italy Poland Spain

Glossary

  • Epilepsy: A brain condition that causes repeated seizures.
  • Focal seizure: A seizure that starts in one part of the brain.
  • Primary generalized tonic-clonic seizure: A seizure that affects both sides of the brain from the start and usually causes stiffening and shaking.
  • Focal epilepsy: Epilepsy where seizures begin in one area of the brain.
  • Phase 3: A later stage of clinical research with more participants, used to study how well a treatment works and how safe it is.
  • Open-label extension: A follow-up study where both the researchers and participants know what treatment is being given.
  • Double-blind: A study design where neither the participant nor the researcher knows who gets the study drug or placebo.
  • Randomized: Participants are assigned to treatment groups by chance.
  • Placebo: An inactive treatment used for comparison in a study.
  • Treatment-emergent adverse events (TEAEs): Health problems that start or get worse after treatment begins.
  • Electrocardiogram (ECG): A test that records the heart's electrical activity.
  • 50% responder: A person who has at least a 50% reduction in seizure frequency during the treatment period.

References

  1. https://clinicaltrials.gov/study/2025-521640-38-00
  2. https://clinicaltrials.gov/study/2025-524038-24-00