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

Phase 3

Recruiting

• Conditions: Developmental and Epileptic Encephalopathy 1
• Interventions: Drug: 1.0mg/kg/day PRAX-562; Drug: Placebo; Drug: 1.5mg/kg/day PRAX-562

• Registration Number: NCT07010471
• Lead Sponsor: Praxis Precision Medicines

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-05-30
• Study First Submitted QC: 2025-05-30
• Study First Posted: 2025-06-08
• Status Verified: 2025-07
• Study Start: 2025-07-09
• Last Update Submitted: 2025-07-31
• Last Update Posted: 2025-08-05
• Primary Completion: 2026-04
• Study Completion: 2027-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 160

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Part B: Open-Label Extension Treatment Period	1.0mg/kg/day PRAX-562	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
Part B: Open-Label Extension Treatment Period	1.5mg/kg/day PRAX-562	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
Part A: Double-Blind Treatment Period	1.0mg/kg/day PRAX-562	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
Part A: Double-Blind Treatment Period	1.5mg/kg/day PRAX-562	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
Part A: Double-Blind Treatment Period (Placebo)	Placebo	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

Primary Outcome Measures

Name	Time	Method
To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo	16 weeks	Change from baseline in monthly motor seizure frequency

Secondary Outcome Measures

Name	Time	Method
Achieve >50% reduction in monthly seizure frequency from baseline	16 weeks	Proportion of patients achieving \>50% reduction in monthly seizure frequency from baseline
Change in seizure-free days	16 weeks	Change in seizure-free days from baseline
Clinical Global Impression-Severity questionnaire	16 weeks	CGI-S at baseline after 16 weeks
Clinical Global Impression-Improvement questionnaire	16 weeks	CGI-I subdomains scores after 16 weeks
Caregiver Global Impression-Severity questionnaire	16 weeks	CgGI-S at baseline after 16 weeks
Caregiver Global Impression-Improvement questionnaire	16 weeks	CgGI-I subdomains scores after 16 weeks
To evaluate the safety and tolerability of relutrigine in participants with DEEs	16 weeks	Incidence and severity of TEAEs
Columbia-Suicide Severity Rating Scale questionnaire	16 weeks	Incidence of suicidal ideation or behavior as measured by the C-SSRS
Evaluate the safety and tolerability of relutrigine in participants with DEEs	16 weeks	Incidence of clinically significant ECG abnormalities
To evaluate the safety labs and tolerability of relutrigine in participants with DEEs	16 weeks	The principal investigator (PI) or sub investigator will review the laboratory report and document this review. Any clinically significant adverse changes occurring during the clinical trial will be documented as adverse events.
To evaluate the change in respiratory rate and tolerability of relutrigine in participants with DEEs	16 weeks	Change in respiratory rate in breaths per minute
To evaluate the change in blood pressure and tolerability of relutrigine in participants with DEEs	16 weeks	Change in blood pressure in mm/Hg
To evaluate the change in pulse and tolerability of relutrigine in participants with DEEs	16 weeks	Change in heart rate in beats per minute
To evaluate the change in body temperature and tolerability of relutrigine in participants with DEEs	16 weeks	Change in tympanic temperature in Celsius

Trial Locations

Locations (1)

Praxis Research Site; 🇺🇸 Chevy Chase, Maryland, United States