A Clinical Trial for Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine

NCT07010471 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PRAX-562-311
• Study Type: interventional
• Target: 160
• Locations: 2 countries
• Sites: 5

Brief Summary

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 Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension

Conditions

Developmental and Epileptic Encephalopathy 1

Keywords

DEE; Developmental and Epileptic Encephalopathy; epilepsy

Outcome Measures

Primary Outcomes (1)

• To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo

  Change from baseline in monthly motor seizure frequency

  16 weeks

Secondary Outcomes (14)

• Achieve >50% reduction in monthly seizure frequency from baseline

  16 weeks

• Change in seizure-free days

  16 weeks

• Clinical Global Impression-Severity questionnaire

  16 weeks

• Clinical Global Impression-Improvement questionnaire

  16 weeks

• Caregiver Global Impression-Severity questionnaire

  16 weeks

Study Arms (3)

Part A: Double-Blind Treatment Period

EXPERIMENTAL

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

Drug: 1.0mg/kg/day PRAX-562; Drug: 1.5mg/kg/day PRAX-562

Part A: Double-Blind Treatment Period (Placebo)

PLACEBO COMPARATOR

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

Drug: Placebo

Part B: Open-Label Extension Treatment Period

EXPERIMENTAL

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

Drug: 1.0mg/kg/day PRAX-562; Drug: 1.5mg/kg/day PRAX-562

Interventions

1.0mg/kg/day PRAX-562 DRUG

Once daily orally or gastronomy/jejunostomy

Also known as: relutrigine

Part A: Double-Blind Treatment Period; Part B: Open-Label Extension Treatment Period

1.5mg/kg/day PRAX-562 DRUG

Once daily orally or gastronomy/jejunostomy

Part A: Double-Blind Treatment Period; Part B: Open-Label Extension Treatment Period

Placebo DRUG

Once daily orally or gastronomy/jejunostomy

Part A: Double-Blind Treatment Period (Placebo)

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Praxis Precision Medicines: lead

Study Sites (5)

Praxis Research Site

La Jolla, California, 92037, United States

RECRUITING

Praxis Research Site

Gulf Breeze, Florida, 32561, United States

RECRUITING

Praxis Research Site

Chevy Chase, Maryland, 20815, United States

RECRUITING

Praxis Research Site

Roseville, Minnesota, 55113, United States

RECRUITING

Praxis Research Site

São Paulo, 15090, Brazil

RECRUITING

MeSH Terms

Conditions

Infantile Epileptic-Dyskinetic Encephalopathy; Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Medical Director

  Praxis Precision Medicines

  STUDY DIRECTOR

Central Study Contacts

Head of Pharmacovigilance

CONTACT

617-300-8460; clinicaltrials@praxismedicines.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 30, 2025
• First Posted: June 8, 2025
• Study Start: July 9, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: December 18, 2025

Record last verified: 2025-12

Locations

US (4); BR (1)