PRAX-562 for Epilepsy

(EMBOLD Trial)

Recruiting at 6 trial locations

Overseen By Head of Pharmacovigilance

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Praxis Precision Medicines

Must not be taking: Sodium channel blockers

Disqualifiers: Other genetic variants, Status epilepticus, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 16 weeks

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a Phase 2, double-blind, randomized clinical trial to explore the safety, tolerability, efficacy, and pharmacokinetics of PRAX-562 in pediatric participants who have seizures associated with early-onset SCN2A-DEE and SCN8A-DEE.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team for guidance.

Who Is on the Research Team?

Medical Director

Principal Investigator

Praxis Precision Medicines

Are You a Good Fit for This Trial?

This trial is for children with rare genetic variants causing seizures within the first months of life. They must have frequent motor seizures, but can't join if they have other significant genetic disorders or a history of seizure worsening due to sodium channel blockers.

Inclusion Criteria

I've had at least 8 motor seizures in the last 4 weeks and during a 28-day observation period.

Additional inclusion criteria apply and will be assessed by the study team

I have a rare SCN2A variant with seizures starting in the first 3 months, or a new SCN8A variant with seizures in the first 6 months.

Exclusion Criteria

Additional exclusion criteria apply and will be assessed by the study team

I have a genetic variant linked to my epilepsy or developmental disorder, not related to SCN2A or SCN8A genes.

I've been hospitalized and intubated for seizures at least twice in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either 1.0 mg/kg/day or 0.5 mg/kg/day PRAX-562 for 16 weeks, with some receiving placebo for the last 4 weeks

16 weeks

Regular visits for monitoring and PK sampling at Weeks 2, 4, 6, 8, 10, 12, and 16

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Eligible participants receive 1.0 mg/kg/day or 0.5 mg/kg/day PRAX-562 for up to 144 weeks

Up to 144 weeks

Visits at Day 1, Week 16, Week 32, and Week 48 for efficacy assessments

What Are the Treatments Tested in This Trial?

Interventions

• PRAX-562

Trial Overview The study tests PRAX-562's safety and effectiveness in treating seizures associated with DEE. It involves different parts: some get the drug for up to 16 weeks, while others receive it alongside a placebo part of the time.

How Is the Trial Designed?

Group I: Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562 Experimental Treatment 1 Intervention

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

Group II: Part A: Randomized, Double-Blind 1.0 mg/kg/day PRAX-562 or PRAX-562/Placebo Experimental Treatment 1 Intervention

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 III: Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo Experimental Treatment 1 Intervention

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 IV: Open-Label Extension Treatment 1.0 mg/kg/day PRAX-562 Experimental Treatment 1 Intervention

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

PRAX-562 is already approved in United States, European Union for the following indications:

Approved in United States as PRAX-562 for:

• Developmental and Epileptic Encephalopathies (DEE) associated with SCN2A and SCN8A mutations

Approved in European Union as PRAX-562 for:

• Developmental and Epileptic Encephalopathies (DEE) associated with SCN2A and SCN8A mutations

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+