A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
77
Once daily oral or G-tube treatment.
Praxis Research Site
Atlanta, Georgia, United States
Praxis Research Site
Chicago, Illinois, United States
Praxis Research Site
Minneapolis, Minnesota, United States
Praxis Research Site
Hackensack, New Jersey, United States
PART A (Cohorts 1 and 2) RDB: To evaluate the safety and tolerability of PRAX 562 in pediatric participants with SCN2A- and SCN8A- DEEs
Changes from baseline in monthly (28-day) motor seizure frequency
Time frame: 16 weeks
PART B (Cohorts 1 and 2) OLE: To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs
Incidence and severity of TEAEs
Time frame: 48 weeks
PART A (Cohorts 1 and 2) RDB: To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs
Changes from baseline in monthly (28-day) motor seizure frequency
Time frame: 16 weeks
Plasma concentrations of PRAX-562
Sparse pharmacokinetic (PK) sampling will be used to calculate mean concentrations at baseline, and at Weeks 2, 4, 6, 8,10, 12 and 16.
Time frame: 16 weeks
Seizure Frequency (OLE Extension)
Efficacy assessments (seizure diary) will be collected daily and reviewed at timepoints Day 1, Week 16, Week 32, and Week 48.
Time frame: 48 weeks
To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs
Changes from baseline in monthly (28-day) motor seizure frequency
Time frame: 16 weeks
Incidence of Treatment-Emergent Adverse Events (TEAEs) [Safety and Tolerability])
The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
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Praxis Research Site
Tel Litwinsky, Israel
Praxis Research Site
Madrid, Spain
Praxis Research Site
Glasgow, United Kingdom
Praxis Research Site
London, United Kingdom
Time frame: 16 weeks