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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-13

Study Completion Date

2027-09-30

Brief Summary

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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.

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Detailed Description

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Conditions

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SCN2A-DEE Epilepsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

No masking in the 2 open-label parts, masking as indicated in the 2 double-blind, randomized parts.

Study Groups

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Preliminary Safety

Open-label PRAX-222

Group Type EXPERIMENTAL

PRAX-222 - Initial Dose

Intervention Type DRUG

PRAX-222

Dose Escalation - PRAX-222

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

Group Type EXPERIMENTAL

PRAX-222 - Initial Ascending Doses

Intervention Type DRUG

Ascending doses of PRAX-222

Dose Escalation - Placebo

Double-blind placebo procedure

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type PROCEDURE

Placebo procedure

Optional Dose Escalation - PRAX-222

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

Group Type EXPERIMENTAL

PRAX-222 - Optional Ascending Doses

Intervention Type DRUG

Escalation of PRAX-222 dose(s)

Optional Dose Escalation - Placebo

Double-blind placebo procedure

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type PROCEDURE

Placebo procedure

Confirmatory Dosing - PRAX-222

Double-blind fixed-dose PRAX-222

Group Type EXPERIMENTAL

PRAX-222 - Fixed Doses

Intervention Type DRUG

Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222

Confirmatory Dosing - Placebo

Double-blind placebo procedure

Group Type EXPERIMENTAL

Placebo

Intervention Type PROCEDURE

Placebo procedure

Open-label PRAX-222

Group Type EXPERIMENTAL

PRAX-222 - Fixed Doses

Intervention Type DRUG

Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222

Interventions

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PRAX-222 - Initial Dose

PRAX-222

Intervention Type DRUG

PRAX-222 - Initial Ascending Doses

Ascending doses of PRAX-222

Intervention Type DRUG

PRAX-222 - Optional Ascending Doses

Escalation of PRAX-222 dose(s)

Intervention Type DRUG

PRAX-222 - Fixed Doses

Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222

Intervention Type DRUG

Placebo

Placebo procedure

Intervention Type PROCEDURE

Eligibility Criteria

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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.

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

Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No