Clinical trial NCT07010471: Randomized, Double-Blind Study in Participants With DEE to Assess Efficacy, Safety, Tolerability, and PK of Relutrigine
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Developmental and Epileptic Encephalopathy 1
Keywords: DEE
Developmental and Epileptic Encephalopathy
epilepsy
Lead sponsor: Praxis Precision Medicines
Sponsor class: INDUSTRY
First posted: 2025-06-08
Enrollment: 160
Sex: ALL
Minimum age (years): 2
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo
timeFrame: 16 weeks
Secondary outcomes:
measure: To explore the effect of relutrigine on additional efficacy outcomes in participants with DEEs
timeFrame: 16 weeks
measure: To explore the effect of relutrigine on additional efficacy outcomes in participants with DEEs
timeFrame: 16 weeks
measure: To explore the effect of relutrigine on additional efficacy outcomes in participants with DEEs
timeFrame: 16 weeks
measure: To explore the effect of relutrigine on additional efficacy outcomes in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
measure: To evaluate the safety and tolerability of relutrigine in participants with DEEs
timeFrame: 16 weeks
Other outcomes:
Locations:
city: Chevy Chase
country: United States
facility: Praxis Research Site
state: Maryland
Study officials:
affiliation: Praxis Precision Medicines
name: Medical Director
role: STUDY_DIRECTOR
References: