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

Launched by PRAXIS PRECISION MEDICINES · May 30, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called Relutrigine, aimed at helping people with Developmental and Epileptic Encephalopathy, a serious condition that causes developmental delays and seizures. The trial is in its third phase, which means it is testing the drug's effectiveness, safety, and how well it is tolerated by participants. It will involve multiple centers and will compare Relutrigine to a placebo (a dummy treatment) to see how well it works. The trial is not yet recruiting participants, but it is open to individuals aged 2 years to 66 years who have been diagnosed with this condition and started having seizures before they turned 12 years old.

To be eligible for the trial, participants need to weigh more than 7 kg and have a documented diagnosis of Developmental and Epileptic Encephalopathy. However, certain health issues, such as serious heart problems or recent severe seizures, may prevent someone from participating. Those who join the study will receive either the medication or a placebo and will be closely monitored throughout the trial. It's important to note that the study will also include an open-label extension, where everyone may receive the actual medication after the initial phase. If you or your loved one is interested in participating, it's best to discuss this with a healthcare provider for more personalized information.

Gender

ALL

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.