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

ConditionDevelopmental and Epileptic Encephalopathy 1
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-65
SponsorPraxis Precision Medicines

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • 1.0mg/kg/day PRAX-562

    Drug

    Once daily orally or gastronomy/jejunostomy

  • 1.5mg/kg/day PRAX-562

    Drug

    Once daily orally or gastronomy/jejunostomy

  • Placebo

    Drug

    Once daily orally or gastronomy/jejunostomy

Treatment groups

160 Participants
are divided into 3 treatment groups
Group A: Part A: Double-Blind Treatment PeriodExperimental treatment 2 interventions
Group B: Part A: Double-Blind Treatment Period (Placebo)Placebo comparator 1 intervention
Group C: Part B: Open-Label Extension Treatment PeriodExperimental treatment 2 interventions

Trial outcomes

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

Time frame
16 weeks

Secondary outcomes

1

Achieve >50% reduction in monthly seizure frequency from baseline

Proportion of patients achieving \>50% reduction in monthly seizure frequency from baseline

Time frame
16 weeks
2

Change in seizure-free days

Change in seizure-free days from baseline

Time frame
16 weeks
3

Clinical Global Impression-Severity questionnaire

CGI-S at baseline after 16 weeks

Time frame
16 weeks
4

Clinical Global Impression-Improvement questionnaire

CGI-I subdomains scores after 16 weeks

Time frame
16 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Praxis Precision Medicines

Lead sponsor

Source ClinicalTrials.gov
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