Praxis Precision Medicines, Inc. ((PRAX)) announced an update on their ongoing clinical study.
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Praxis Precision Medicines, Inc. is conducting a clinical trial titled ‘A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy.’ The study aims to evaluate the efficacy and safety of Elsunersen in treating pediatric patients with SCN2A-related epileptic encephalopathy, a severe neurological disorder.
The intervention being tested is Elsunersen, administered intrathecally every four weeks for 24 weeks, with two dosage levels: 1mg and 0.5mg. A sham procedure is used as a comparator in the double-blind phase.
The study is designed as a randomized, sequential, and quadruple-masked trial, primarily focused on treatment. It includes three cohorts, with the first cohort being masked and subsequent cohorts open-label, contingent on meeting specific milestones.
The study began on June 5, 2025, with primary completion expected soon after. The latest update was submitted on October 1, 2025, indicating ongoing recruitment.
This clinical update could influence Praxis Precision Medicines’ stock performance positively, as successful results might enhance investor confidence. The competitive landscape in the treatment of rare neurological disorders remains dynamic, with several companies vying for breakthroughs.
The study is ongoing, with further details available on the ClinicalTrials portal.