Relutrigine treatment for people with Developmental and Epileptic Encephalopathy (DEE)

A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label Extension

This Phase 3 study will test whether two doses of relutrigine reduce seizures and are safe and tolerable for people with Developmental and Epileptic Encephalopathy.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 160 (estimated)
• Ages: 2 Years to 65 Years
• Sex: All
• Sponsor: Praxis Precision Medicines (industry)
• Locations: 11 sites (La Jolla, California and 10 other locations)
• Trial ID: NCT07010471 on ClinicalTrials.gov

What this trial studies

This is a randomized, multi-center, double-blind, placebo-controlled Phase 3 trial comparing two weight-based doses of relutrigine (1.0 mg/kg/day and 1.5 mg/kg/day) against placebo, followed by an open-label extension where participants can receive active drug. Participants must have a documented DEE diagnosis with seizure onset before age 12 and meet basic safety criteria including weight >7 kg and acceptable ECG results. The trial includes safety monitoring, tolerability assessments, and pharmacokinetic sampling to characterize drug levels, along with efficacy endpoints related to seizure frequency and severity. Sites are run by Praxis Precision Medicines at three U.S. locations.

Who should consider this trial

Good fit: Ideal candidates are people with a documented developmental and epileptic encephalopathy with seizure onset before age 12 who meet ECG and safety criteria and weigh more than 7 kg.

Not a fit: People with certain cardiac rhythm disorders, a recent history of severe convulsive status epilepticus, unstable nerve stimulation devices, or recent participation in other investigational drug studies may not be eligible or derive benefit.

Why it matters

Potential benefit: If successful, relutrigine could reduce seizure frequency and improve clinical outcomes for people with DEE.

How similar studies have performed: Early-phase studies of PRAX-562 (relutrigine) have shown promising safety and seizure-reduction signals in smaller or earlier cohorts, but large randomized Phase 3 evidence in DEE is still limited.

Eligibility criteria

Inclusion Criteria:

* Has a documented diagnosis of a developmental and epileptic encephalopathy.
* Onset of seizures \<12 years old.
* Has a weight \>7 kg at the time of signing consent/assent.

Exclusion Criteria:

* Has a history of left bundle branch block, arrhythmias, Brugada syndrome, congenital heart disease, familial short QT syndrome, or family history of sudden death or ventricular arrhythmias, including idiopathic ventricular fibrillation.
* Had 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
* 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.
* Any nerve stimulation must have been placed at least 3 months prior to Screening with at least 1 month of stable settings prior to Screening.
* Has received any other experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy.
* Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within 5 half-lives of the last study drug dose.

Where this trial is running

La Jolla, California and 10 other locations

• Praxis Research Site — La Jolla, California, United States (RECRUITING)
• Praxis Research Site — Gulf Breeze, Florida, United States (RECRUITING)
• Praxis Research Site — Chevy Chase, Maryland, United States (RECRUITING)
• Praxis Research Site — Roseville, Minnesota, United States (RECRUITING)
• Praxis Research Site — Hackensack, New Jersey, United States (RECRUITING)
• Praxis Research Site — Philadelphia, Pennsylvania, United States (RECRUITING)
• Praxis Research Site — Heidelberg, Victoria, Australia (RECRUITING)
• Praxis Research Site — Rio Grande, Do Sul, Brazil (RECRUITING)
• Praxis Research Site — Paraná, Brazil (RECRUITING)
• Praxis Research Site — Rio de Janeiro, Brazil (RECRUITING)
• Praxis Research Site — São Paulo, Brazil (RECRUITING)

Study contacts

• Study coordinator: Head of Pharmacovigilance

Conditions: Developmental and Epileptic Encephalopathy 1, DEE, Developmental and Epileptic Encephalopathy, epilepsy