Can a new drug tame severe childhood epilepsy?

NCT ID NCT05737784

Ongoing Disease control Sponsor: Praxis Precision Medicines Source: ClinicalTrials.gov ↗

First seen Aug 03, 2026 · Last updated Aug 04, 2026 · Updated 1 time

Summary

This trial is testing an experimental drug called PRAX-222 in children aged 2 to 18 who have a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug is safe and can reduce how often seizures happen. Participants will receive either PRAX-222 or a placebo, and their seizure frequency will be tracked over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: PRAX-222
What this could lead to: If successful, this could lead to a targeted treatment that reduces seizures in children with a rare, severe form of epilepsy caused by SCN2A gene changes.
What could go wrong: This is an early-stage trial, so the drug may not work as hoped or may cause side effects. The results may not apply to all children with SCN2A-related epilepsy.

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Conditions

The condition(s) this trial relates to.

developmental and epileptic encephalopathy, 11 epilepsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

EPILEPSY SCN2A-DEE

Contacts and locations

Locations

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil
Le Bonheur Childrens Hospital

Memphis, Tennessee, 38103, United States
Praxis Research Site

São Paulo, 05403-010, Brazil

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