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

Praxis Precision Medicines

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: SCN2A-DEE; Epilepsy

Treatments: Drug: PRAX-222 - Initial Dose; Drug: PRAX-222 - Fixed Doses; Drug: PRAX-222 - Optional Ascending Doses; Procedure: Placebo; Drug: PRAX-222 - Initial Ascending Doses

Study type: Interventional

Funder types: Industry

Identifiers

NCT05737784

PRAX-222-111

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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60 participants in 8 patient groups, including a placebo group

Preliminary Safety

Experimental group

Description:

Open-label PRAX-222

Treatment:

Drug: PRAX-222 - Initial Dose

Dose Escalation - PRAX-222

Experimental group

Description:

Initial dose escalation consisting of double-blind ascending doses of PRAX-222

Treatment:

Drug: PRAX-222 - Initial Ascending Doses

Dose Escalation - Placebo

Placebo Comparator group

Description:

Double-blind placebo procedure

Treatment:

Procedure: Placebo

Optional Dose Escalation - PRAX-222

Experimental group

Description:

Optional dose escalation consisting of double-blind ascending doses of PRAX-222

Treatment:

Drug: PRAX-222 - Optional Ascending Doses

Optional Dose Escalation - Placebo

Placebo Comparator group

Description:

Double-blind placebo procedure

Treatment:

Procedure: Placebo

Confirmatory Dosing - PRAX-222

Experimental group

Description:

Double-blind fixed-dose PRAX-222

Treatment:

Drug: PRAX-222 - Fixed Doses

Confirmatory Dosing - Placebo

Experimental group

Description:

Double-blind placebo procedure

Treatment:

Procedure: Placebo

Open-label PRAX-222

Experimental group

Description:

Open-label PRAX-222

Treatment:

Drug: PRAX-222 - Fixed Doses