In short
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.
Key points
- VORMATRIGINE is being studied in two authorised Phase 3 clinical trials in adults with epilepsy. One trial is an open-label extension focused on safety and tolerability in adults with focal onset seizures or primary generalized tonic-clonic seizures. The other is a double-blind, randomized trial in adults with focal epilepsy who are taking 1 to 3 anti-seizure medicines. The main goals are to check seizure reduction, side effects, vital signs, lab results, ECG changes, and suicidality. The studies plan to enroll 196 and 300 participants, respectively.
Clinical trials overview
Two Phase 3 trials are investigating VORMATRIGINE in adults with epilepsy. Both studies are interventional, which means participants receive a study treatment and the researchers measure the results.
The trials are authorised and together focus on adults with focal seizures, focal epilepsy, and primary generalized tonic-clonic seizures. The studies are designed to look at both how well the treatment may work and how safe it is in these groups.
Trial 1: Open-label extension in adults with epilepsy
The first study is an open-label extension clinical trial in adult patients with epilepsy. It includes adults with focal onset seizures or primary generalized tonic-clonic seizures.
This trial plans to enroll 196 people and is currently authorised. Its brief summary says the goal is to assess the safety and tolerability of VORMATRIGINE in these adults.
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. 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).
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. In a double-blind study, neither the participant nor the researcher knows who gets the study drug or placebo during the trial.
This trial plans to enroll 300 adults and is also authorised. 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).
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. This is a common way to measure seizure improvement in epilepsy studies.
Key endpoints and what they mean
Clinical trials use endpoints to decide what result they want to measure. In these studies, the endpoints are focused on seizure control, safety, and overall tolerability.
- Safety: whether the treatment causes health problems that matter during the trial.
- Tolerability: how well participants are able to stay on the treatment without problems that lead to stopping it.
- Seizure frequency: how often seizures happen over time.
- Vital signs: basic body measurements such as blood pressure and pulse.
- Laboratory results: blood or other test results checked during the study.
- ECG parameters: measurements from a heart tracing test.
Who may participate
These trials are for adults with epilepsy, not children. 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.
The source data do not give full details about all inclusion or exclusion rules, so the exact entry criteria are not fully listed here.
Trial design terms explained
Open-label extension means a follow-up study where the treatment is known to everyone in the study. This type of study often helps collect longer-term safety information.
Randomized means participants are assigned by chance to a study group. Placebo means an inactive treatment used for comparison, so researchers can see whether the study drug works better than no active treatment.
Interventional means the study actively gives a treatment and measures what happens next. Phase 3 means the research is in a later stage and usually includes more participants than early studies.