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A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Purpose

The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.

Conditions

SCN2A-DEE
Epilepsy

Eligibility

Eligible Ages: Between 2 Years and 18 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion Criteria

Has onset of seizures prior to 3 months of age. - Has a minimum weight of at least 10 kg at screening. - Has a documented SCN2A variant through genetic testing obtained via a laboratory accredited per Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) or equivalent. - Additional inclusion criteria apply and will be assessed by the study team

Exclusion Criteria

Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder. - Is taking more than 2 sodium channel blocking anti-seizure medications - Additional exclusion criteria apply and will be assessed by the study team

Study Design

Phase: Phase 1/Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Seamless study design with sequential study parts, including an open-label safety part; a double-blind, randomized dose-escalation part; a double-blind, randomized confirmatory part; and an open-label extension part.
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: No masking in the 2 open-label parts, masking as indicated in the 2 double-blind, randomized parts.

Arm Groups

Arm	Description	Assigned Intervention
Experimental Preliminary Safety	Open-label PRAX-222	Drug: PRAX-222 - Initial Dose PRAX-222
Experimental Dose Escalation - PRAX-222	Initial dose escalation consisting of double-blind ascending doses of PRAX-222	Drug: PRAX-222 - Initial Ascending Doses Ascending doses of PRAX-222 Drug: PRAX-222 - Optional Ascending Doses Escalation of PRAX-222 dose(s)
Placebo Comparator Dose Escalation - Placebo	Double-blind placebo procedure	Procedure: Placebo Placebo procedure
Experimental Optional Dose Escalation - PRAX-222	Optional dose escalation consisting of double-blind ascending doses of PRAX-222	Drug: PRAX-222 - Optional Ascending Doses Escalation of PRAX-222 dose(s)
Placebo Comparator Optional Dose Escalation - Placebo	Double-blind placebo procedure	Procedure: Placebo Placebo procedure
Experimental Confirmatory Dosing - PRAX-222	Double-blind fixed-dose PRAX-222	Drug: PRAX-222 - Fixed Doses Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222
Experimental Confirmatory Dosing - Placebo	Double-blind placebo procedure	Procedure: Placebo Placebo procedure
Experimental Open-label PRAX-222	Open-label PRAX-222	Drug: PRAX-222 - Fixed Doses Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222

Recruiting Locations

More Details

NCT ID: NCT05737784
Status: Recruiting
Sponsor: Praxis Precision Medicines

Study Contact

Head of Pharmacovigilance
617-300-8460
clinicaltrials@praxismedicines.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.