PRAX-222 for Epileptic Encephalopathy

Recruiting at 1 trial location

Overseen By Head of Pharmacovigilance

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Praxis Precision Medicines

Must not be taking: Sodium channel blockers

Disqualifiers: Other genetic variants, others

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

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you cannot be on more than 2 sodium channel blocking anti-seizure medications.

Research Team

Medical Director

Principal Investigator

Praxis Precision Medicines

Eligibility Criteria

This trial is for children aged 2-18 with early onset SCN2A developmental and epileptic encephalopathy, who had seizures start before 3 months old, weigh at least 10 kg, and have a specific genetic variant. Kids can't join if they have other significant genetic changes or are on more than two sodium channel blocking seizure meds.

Inclusion Criteria

I weigh at least 10 kg.

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

I have a confirmed SCN2A variant from an accredited lab test.

Exclusion Criteria

My epilepsy or developmental disorder is not caused by known genetic changes outside of the SCN2A gene.

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

I am taking more than two medications for seizures that block sodium channels.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Preliminary Safety

Open-label PRAX-222 administration to assess initial safety and seizure frequency

12 to 16 weeks

Dose Escalation

Double-blind ascending doses of PRAX-222 or placebo to determine optimal dosing

24 to 28 weeks

Confirmatory Dosing

Double-blind fixed-dose PRAX-222 or placebo to confirm efficacy and safety

60 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• PRAX-222

Trial Overview The study tests PRAX-222's effects in various doses compared to a placebo. It starts with an initial dose of PRAX-222, followed by increasing amounts if safe, and then settles on fixed doses to see how well it works for these young epilepsy patients.

Participant Groups

Group I: Preliminary Safety Experimental Treatment 1 Intervention

Open-label PRAX-222

Group II: Optional Dose Escalation - PRAX-222 Experimental Treatment 1 Intervention

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

Group III: Open-label PRAX-222 Experimental Treatment 1 Intervention

Open-label PRAX-222

Group IV: Dose Escalation - PRAX-222 Experimental Treatment 1 Intervention

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

Group V: Confirmatory Dosing - Placebo Experimental Treatment 1 Intervention

Double-blind placebo procedure

Group VI: Confirmatory Dosing - PRAX-222 Experimental Treatment 1 Intervention

Double-blind fixed-dose PRAX-222

Group VII: Dose Escalation - Placebo Placebo Group 1 Intervention

Double-blind placebo procedure

Group VIII: Optional Dose Escalation - Placebo Placebo Group 1 Intervention

Double-blind placebo procedure

PRAX-222 is already approved in European Union, United States for the following indications:

Approved in European Union as Elsunersen for:

• SCN2A Gain of Function developmental and epileptic encephalopathy (DEE)

Approved in United States as Elsunersen for:

• SCN2A Gain of Function developmental and epileptic encephalopathy (DEE)

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+