A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

NCT ID: NCT05818553

Last Updated: 2026-01-29

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

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

77 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-02

Study Completion Date

2027-03-01

Brief Summary

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A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

Detailed Description

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A Phase 2, double-blind, randomized clinical trial to evaluate the safety and tolerability of PRAX 562 in pediatric participants with SCN2A- and SCN8A- DEEs.

Conditions

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SCN2A Encephalopathy SCN8A Encephalopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Parallel group
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Double-blind

Study Groups

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Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo

Eligible participants from each cohort will be randomized in a 1:1 ratio to either 0.5 milligrams/kilograms/day (mg/kg/day) PRAX-562 for 16 weeks (PRAX-562 arm) or 0.5 mg/kg/day PRAX-562 for 12 weeks and matching placebo for 4 weeks (PRAX-562/placebo arm) administered orally or via gastrostomy tube (G-tube).

Group Type EXPERIMENTAL

PRAX-562

Intervention Type DRUG

Once daily oral or G-tube treatment.

Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562

Eligible participants will receive 0.5mg/kg/day administered orally or via G-tube for up to 144 weeks.

Group Type EXPERIMENTAL

PRAX-562

Intervention Type DRUG

Once daily oral or G-tube treatment.

Part A: Randomized, Double-Blind 1.0 mg/kg/day PRAX-562 or PRAX-562/Placebo

Eligible participants from each cohort will be randomized in a 1:1 ratio to either 1.0 milligrams/kilograms/day (mg/kg/day) PRAX-562 for 16 weeks (PRAX-562 arm) or 1.0 mg/kg/day PRAX-562 for 12 weeks and matching placebo for 4 weeks (PRAX-562/placebo arm) administered orally or via gastrostomy tube (G-tube).

Group Type EXPERIMENTAL

PRAX-562

Intervention Type DRUG

Once daily oral or G-tube treatment.

Open-Label Extension Treatment 1.0 mg/kg/day PRAX-562

Eligible participants will receive 1.0 mg/kg/day administered orally or via G-tube for up to 144 weeks.

Group Type EXPERIMENTAL

PRAX-562

Intervention Type DRUG

Once daily oral or G-tube treatment.

Interventions

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PRAX-562

Once daily oral or G-tube treatment.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Has a documented variant in SCN2A with onset of seizures occurring in the first 3 months of life or has a diagnosis of SCN8A-DEE supported by both clinical and genetic findings.
* Has a seizure frequency as follows:

* At least 8 countable motor seizures in the 4 weeks immediately prior to Screening as reported by the parent/legal guardian or in the opinion of the investigator as documented in medical notes.

AND o At least 8 countable motor seizures during the 28 day Baseline Observation Period (during which seizure frequency is recorded in a daily seizure diary).

Exclusion Criteria

* Has any clinically significant or known pathogenic or likely pathogenic genetic variant other than in SCN2A and SCN8A or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
* Has a documented, functionally characterized loss-of-function (LoF) missense variant or a presumed LoF variant (nonsense or frameshift variant) based on genetic testing and/or clinical evidence that prior exposure to a sodium channel blocker (SCB) medication worsened seizures.
* Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
Minimum Eligible Age

1 Year

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Praxis Precision Medicines

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Praxis Precision Medicines

Locations

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Praxis Research Site

Atlanta, Georgia, United States

Site Status

Praxis Research Site

Chicago, Illinois, United States

Site Status

Praxis Research Site

Minneapolis, Minnesota, United States

Site Status

Praxis Research Site

Hackensack, New Jersey, United States

Site Status

Praxis Research Site

Tel Litwinsky, , Israel

Site Status

Praxis Research Site

Madrid, , Spain

Site Status

Praxis Research Site

Glasgow, , United Kingdom

Site Status

Praxis Research Site

London, , United Kingdom

Site Status

Countries

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United States Israel Spain United Kingdom

Other Identifiers

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PRAX-562-221

Identifier Type: -

Identifier Source: org_study_id

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