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Small Molecule

PRAX-562 for Epilepsy (EMBOLD Trial)

Phase 2
Recruiting
Research Sponsored by Praxis Precision Medicines
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has a documented rare missense variant in SCN2A with onset of seizures occurring in the first three months of life or has a documented de novo (not observed in either parent) missense variant in SCN8A with onset of seizures occurring in the first six months of life
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 48 weeks
Awards & highlights

EMBOLD Trial Summary

This trial will explore if a new medicine is safe, tolerated and effective for children with early-onset seizures.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects are not listed here, common ones may include reactions at the injection site, gastrointestinal issues, changes in behavior or mood, dizziness, and potential allergic reactions.

EMBOLD Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
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.

EMBOLD Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~48 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Treatment-Emergent Adverse Events (TAEs) [Safety and Tolerability])
To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs
Secondary outcome measures
Plasma concentrations of PRAX-562
Seizure Frequency (OLE Extension)
To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs

EMBOLD Trial Design

2Treatment groups
Experimental Treatment
Group I: Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562Experimental Treatment1 Intervention
Eligible participants will receive 0.5mg/kg/day administered orally or via G-tube for 48 weeks.
Group II: Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/PlaceboExperimental Treatment2 Interventions
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).

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Who is running the clinical trial?

Praxis Precision MedicinesLead Sponsor
9 Previous Clinical Trials
1,179 Total Patients Enrolled
Medical DirectorStudy DirectorPraxis Precision Medicines
2,770 Previous Clinical Trials
8,062,029 Total Patients Enrolled

Media Library

PRAX-562 (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT05818553 — Phase 2
Encephalopathy Research Study Groups: Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo, Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562
Encephalopathy Clinical Trial 2023: PRAX-562 Highlights & Side Effects. Trial Name: NCT05818553 — Phase 2
PRAX-562 (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05818553 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How perilous is the 0.5mg/kg/day PRAX-562 dosage during Part B: Open-Label Extension Treatment?

"Taking into account the Phase 2 nature of this study, there is evidence to suggest that Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562 is moderately safe and thus has been rated a score of two by our team at Power."

Answered by AI

Are there any opportunities for the public to partake in this clinical experiment?

"Affirmative, clinicaltrials.gov holds evidence that this medical study began enrolling patients on April 17th 2023 and was recently updated a day later. At present, there is need for up to 20 volunteers at one site."

Answered by AI

Is participation in this research project restricted to persons below a certain age?

"This medical trial is designed for children and adolescents between 2-18 years old. According to the data on clinicaltrials.gov, there are 162 trials available for those younger than 18 and 231 studies catering towards seniors aged 65 or older."

Answered by AI

What is the scale of this experimental treatment?

"Affirmative. According to clinicaltrials.gov, this medical trial is actively recruiting individuals; it was initially listed on April 17th 2023 and most recently updated on the 18th of the same month. This study aims to enrol a total of twenty participants from one site."

Answered by AI

Who meets the qualifications to take part in this experimentation?

"For this research endeavour, 20 minors between the ages of 2 and 18 that are currently afflicted with encephalopathy must be recruited. To qualify for enrolment in the study, participants ought to possess at least eight motor seizures (as outlined by the accompanying note) within four weeks before screening according to a parent/guardian or investigator's account. Furthermore, additional inclusion criteria will be evaluated by the clinical team."

Answered by AI
~6 spots leftby Jun 2024