Relutrigine for Epilepsy

(EMERALD Trial)

Overseen By Head of Pharmacovigilance

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Praxis Precision Medicines

Disqualifiers: Heart disease, Status epilepticus, Pregnancy, others

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

160 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the drug Relutrigine to determine its effectiveness for individuals with developmental and epileptic encephalopathies, conditions that cause severe seizures and affect brain development. The trial compares Relutrigine to a placebo (a sugar pill or treatment with no active drug) to assess its safety and effectiveness. Participants must have begun experiencing seizures before age 12 and weigh more than 7 kg (about 15 pounds). As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Studies have shown that relutrigine is generally safe and well-tolerated by patients. In earlier research, patients taking relutrigine experienced side effects similar to those of other seizure medications, indicating that the side effects were expected and not unusual compared to similar treatments. Even when some patients increased their dose during the study, they still handled the medication well. This suggests that relutrigine could be a dependable option for people with developmental and epileptic encephalopathies. Discuss any concerns with a healthcare provider. ^{1 2 3 4 5}

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

Relutrigine is unique because it offers a new approach to managing epilepsy by potentially delivering more effective seizure control. Unlike standard treatments like valproate or lamotrigine, relutrigine may target the brain's electrical activity differently, possibly providing benefits for patients who don't respond well to existing medications. Additionally, its flexible administration method, which includes oral and gastronomy/jejunostomy options, could improve patient compliance by accommodating different patient needs and preferences. Researchers are particularly excited because this new mechanism and delivery method might lead to better outcomes for individuals with epilepsy.

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

Research has shown that relutrigine can greatly reduce seizures in people with epilepsy. In one study, about 33% of patients taking relutrigine experienced no seizures for over 200 days. Another study found a 46% reduction in seizures compared to those taking a placebo, which contains no active medicine. Some patients also reported feeling more alert and better able to communicate. In the current trial, participants in the open-label extension period will receive relutrigine. Previous research demonstrated a remarkable 90% reduction in seizures. This strong evidence suggests that relutrigine could be an effective treatment for epilepsy. ^{1 2 3 5 6}

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 individuals with Developmental and Epileptic Encephalopathies (DEE), including Dravet syndrome. Participants should be able to take oral medication and comply with study requirements. Specific details about inclusion or exclusion criteria are not provided, but typically these would involve age ranges, severity of condition, and absence of other health issues that could interfere with the study.

Inclusion Criteria

Has a weight >7 kg at the time of signing consent/assent.

I started having seizures before I was 12 years old.

I have been diagnosed with a developmental and epileptic encephalopathy.

Exclusion Criteria

I haven't taken any experimental treatments or drugs in the last 30 days or longer.

I've been hospitalized and intubated for seizures twice in the last 6 months.

Has an abnormal ECG reading, including a QT interval corrected for heart rate using Bazett's method (QTcB) <350 and >450 ms (males), or <360 and >460 ms (females) at Screening and/or on Day 1

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-Blind Treatment

Eligible participants will be randomly assigned in a double-blind manner to receive relutrigine or placebo for 16 weeks

16 weeks

Open-Label Extension

Participants from Part A will have the option to rollover to receive relutrigine for 32 weeks

32 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Relutrigine

Trial Overview The trial is testing Relutrigine at two different doses (1.0mg/kg/day and 1.5mg/kg/day) against a placebo to see if it's effective, safe, tolerable, and how it moves through the body (pharmacokinetics). It's a phase 3 study where participants are randomly assigned to one of the treatments without knowing which one they're getting (double-blind).

How Is the Trial Designed?

Group I: Part B: Open-Label Extension Treatment Period Experimental Treatment 2 Interventions

Participants from Part A will have the option to rollover to Part B to receive 1.0mg-1.5mg/kg of relutrigine once daily orally or gastronomy/jejunostomy for 32 weeks

Group II: Part A: Double-Blind Treatment Period Experimental Treatment 2 Interventions

Eligible participants will be randomly assigned in a double-blind manner and a 1:1 ratio to receive 1.0mg-1.5mg/kg relutrigine or placebo once daily orally or gastronomy/jejunostomy for 16 weeks

Group III: Part A: Double-Blind Treatment Period (Placebo) Placebo Group 1 Intervention

Eligible participants will be randomly assigned in a double-blind manner and a 1:1 ratio to receive 1.0mg-1.5mg/kg relutrigine or placebo once daily orally or gastronomy/jejunostomy for 16 weeks

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-precision-medicines-announces-positive-topline-results-0

Praxis Precision Medicines announces positive topline results ... Over 30% of patients achieved seizure freedom status while on relutrigine. Meaningful gains observed in alertness, communication and seizure severity.

2. aesnet.org aesnet.org/abstractslisting/relutrigine-demonstrates-robust-seizure-reduction-and-seizure-freedom-in-dees-results-from-the-embold-study

relutrigine demonstrates robust seizure reduction and seizure ... At the time of submission, 33% of patients were seizure-free (longest follow-up >200 days), with ~30-70% improvements reported by clinicians and ...

3. praxismedicines.gcs-web.com praxismedicines.gcs-web.com/news-releases/news-release-details/praxis-precision-medicines-receives-fda-breakthrough-therapy

Press Release The results from the open-label extension are remarkable, showing an average of both approximately 90% reduction in seizures and over two months ...

4. scn2a.org scn2a.org/promising-initial-relutrigine-prax-562-clinical-data/

Promising Initial Relutrigine (Prax-562) Clinical Data The trial demonstrated a 46% reduction in seizures compared to placebo, with a safety profile consistent with other drugs in this class.

5. 2minutemedicine.com 2minutemedicine.com/praxis-precisions-relutrigine-reduces-seizures-in-rare-epileptic-disorders-in-phase-2-clinical-trials/

Praxis Precision's relutrigine reduces seizures in rare ... The study showed that patients taking relutrigine experienced a 46% reduction in motor seizures, with 30% of patients achieving seizure freedom.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07010471

A Clinical Trial for Participants With DEE to Assess Efficacy, ... To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo ... To evaluate the safety and tolerability of relutrigine ...