PRAX-562-101

8 evidence topics · 1 source

Objective

Location and study date

No evidence found.

Study design

Parts A and B: ascending-dose study

“randomized, placebo-controlled” (opens the source at this quote in a new tab)

Part C: food-effect study

“open-label, randomized, crossover” (opens the source at this quote in a new tab)

Eligibility criteria

Healthy-adult age range

“healthy adults aged 18-55 years” (opens the source at this quote in a new tab)

Treatment

Dose ranges and food-effect dose

Study part	Dose
Part A, single dose	“2.5 to 150 mg”
Part B, repeated once-daily dosing	“30 to 120 mg”
Part C, fed and fasted dosing	“90 mg”

Study outcomes

Rationale for using safety and tolerability as the primary endpoint

Not applicable.

Statistical analysis description

Number of participants (Planned and analyzed)

Total enrollment

“112 participants were enrolled across the PRAX-562-101 trial (n=88 PRAX-562, n=24 placebo)” (opens the source at this quote in a new tab)

Description of analysis sets

Safety analysis sets: inclusion of all participants

“All participants were included” (opens the source at this quote in a new tab)

Results

Participant disposition

Baseline characteristics

Efficacy results

Not applicable.

Health-related quality of life and patient-reported outcomes

Not applicable.

Safety results

Severe treatment-emergent adverse events

“No severe TEAEs were reported” (opens the source at this quote in a new tab)

Study limitations

Summary

The study enrolled 112 healthy-adult participants rather than participants with SCN2A- or SCN8A-related DEE. Single-dose, repeated-dose, and food-effect findings inform pharmacokinetics and short-term tolerability, but do not establish seizure efficacy, pediatric effectiveness, or long-term safety in the target population.