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

Praxis Precision Medicines

Status and phase: Enrolling; Phase 3

Conditions: Developmental and Epileptic Encephalopathy 1

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT07010471

PRAX-562-311

Details and patient eligibility

About

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

Enrollment: 160 estimated patients

Sex: All

Ages: 2 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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160 participants in 3 patient groups, including a placebo group

Part A: Double-Blind Treatment Period

Experimental group

Description:

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

Treatment:

Drug: 1.0mg/kg/day PRAX-562

Drug: 1.5mg/kg/day PRAX-562

Part A: Double-Blind Treatment Period (Placebo)

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

Part B: Open-Label Extension Treatment Period

Experimental group

Description:

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

Treatment:

Drug: 1.0mg/kg/day PRAX-562

Drug: 1.5mg/kg/day PRAX-562