Vormatrigine for Epilepsy

Enrolling by invitation at 19 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Praxis Precision Medicines

Must be taking: Vormatrigine

Disqualifiers: Pregnancy, Lactation, Other studies, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called vormatrigine to evaluate its effectiveness for adults with epilepsy. Participants will take 30 mg of vormatrigine daily for up to two years to help control seizures. The trial is open to individuals who have previously participated in a vormatrigine study or have taken the medication through special programs, provided they have maintained a good record of their seizures. As a Phase 3 trial, this study represents the final step before FDA approval, giving participants the opportunity to contribute to the potential availability of a new epilepsy treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that you cannot be on any prohibited medication. It's best to discuss your current medications with the trial team to see if they are allowed.

Is there any evidence suggesting that Vormatrigine is likely to be safe for humans?

Research has shown that vormatrigine is generally safe and well-tolerated. Earlier studies found that most patients experienced only mild and temporary side effects, meaning any negative reactions were usually not serious and resolved on their own. Vormatrigine has undergone human testing before, providing reassurance about its safety. While all treatments can have side effects, evidence so far suggests that most people handle vormatrigine well. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for epilepsy?

Most treatments for epilepsy, like valproate or lamotrigine, work by stabilizing electrical activity in the brain or modulating neurotransmitters to prevent seizures. Vormatrigine is unique because it targets specific pathways in the brain that are believed to play a crucial role in seizure activity, potentially offering a more focused approach to treatment. Researchers are excited about vormatrigine because it may provide better control over seizures with fewer side effects, which is a significant improvement over current options. Additionally, its potential long-term effectiveness, with the option to be used for up to two years, could offer sustained relief for patients.

What evidence suggests that Vormatrigine might be an effective treatment for epilepsy?

Research has shown that vormatrigine helps reduce seizures in people with epilepsy. After eight weeks of treatment, patients experienced an average seizure reduction of about 56%. Notably, about 22% of patients had no seizures during this period. These results suggest that vormatrigine can significantly help manage seizures and improve life for those with epilepsy. Participants in this trial will receive 30 mg/day of vormatrigine for up to two years to further evaluate its effectiveness. ^{3 5 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Praxis Precision Medicines

Are You a Good Fit for This Trial?

This trial is for adults with epilepsy who finished a previous vormatrigine study or got the drug through special access. They must have kept good seizure diaries, followed study rules well, and not be planning to join another drug trial or take forbidden meds. Pregnant or breastfeeding individuals can't participate.

Inclusion Criteria

I have completed a clinical trial with vormatrigine or received it through an expanded access program.

Participant who had adequate seizure diary and vormatrigine adherence and had been otherwise adherent to the study procedures in the opinion of the investigator unless deemed unacceptable by sponsor

Exclusion Criteria

Participant who withdrew or discontinued treatment from the treatment period of an eligible clinical trial unless deemed acceptable by the sponsor

I am not planning to join another clinical study or take any forbidden medications.

Participant who is pregnant, lactating or planning to become pregnant

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Treatment

Participants receive 30 mg/day of vormatrigine to assess safety and tolerability

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Vormatrigine

Trial Overview

The trial tests vormatrigine's long-term effects in adults with epilepsy. It's an open-label extension, meaning everyone knows they're getting vormatrigine and there’s no placebo group involved.

How Is the Trial Designed?

Group I: Open-label 30 mg/day vormatrogine for up to 2 years Experimental Treatment 1 Intervention

Participants who meet all eligibility criteria will receive 30 mg of vormatrogine for up to 2 years.

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+

Citations

1.

investors.praxismedicines.com

investors.praxismedicines.com/news-releases/news-release-details/praxis-aes-wrap-best-class-potential-across-rare-pediatric-and/

Praxis AES Wrap-up: Best-in-Class Potential Across Rare ...

Patients taking vormatrigine for 8 weeks on background anti-seizure medications (ASM) saw a 54% median reduction in seizures. In week 1, 58% of ...

2.

praxismedicines.gcs-web.com

praxismedicines.gcs-web.com/news-releases/news-release-details/praxis-precision-medicines-announces-positive-best-disease

Press Release

Dosing with vormatrigine over 8 weeks led to 56.3% median reduction in seizure frequency. Approximately 22% of patients reached 100% ...

3.

clinicaltrialsarena.com

clinicaltrialsarena.com/news/praxis-vormatrigine-eliminates-seizures-in-22-of-patients-in-phase-ii-trial/

Praxis' vormatrigine eliminates seizures in 22% of patients ...

In the study, patients treated with vormatrigine for eight weeks experienced a 56.3% median reduction in seizure frequency. Around 22% of ...

4.

biospace.com

biospace.com/drug-development/praxis-seizure-drug-demonstrates-impressive-efficacy-in-mid-stage-trial

Praxis' Seizure Drug Demonstrates 'Impressive' Efficacy in ...

Over eight weeks of treatment, patients on vormatrigine experienced a 56.3% median decrease in seizure frequency, according to a statement from ...

5.

neurologylive.com

neurologylive.com/view/vormatrigine-reduces-seizures-phase-2-radiant-postpartum-depression-ms-women-ianalumab-produces-significant-phase-3-data-sjrogrens

Vormatrigine Reduces Seizures in Phase 2 RADIANT ...

Over the 8-week treatment period, treatment with 30 mg/day of vormatrigine led to a 56.3% median reduction in seizure frequency, with 54% of ...

6.

praxismedicines.com

praxismedicines.com/wp-content/uploads/2025/08/P248_Petrou_IEC2025_PRAX-628-Ph1-Update.pdf

Background Methods Vormatrigine Continues To Be Well ...

PK and cardiac safety data from PRAX-628-101, a first-in-human Phase ... Vormatrigine demonstrated consistent safety, tolerability, and PK ...

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07287163

Clinical Trial of Vormatrigine in Adult Patients With Epilepsy

An open label extension clinical trial to assess the safety and tolerability of vormatrigine in adults with focal or primary generalized tonic-clonic seizures.

8.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000212741

Updates from the First-in-human Phase 1 Clinical Trial ...

Vormatrigine was well tolerated with AEs mostly mild, transient, and self-resolving. PK data showed dose-dependent exposure proportional to dose ...