Clinical trial NCT05818553: A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)
Official title: A Phase 2,Double-Blind,Randomized Clinical Trial to Explore the Safety,Tolerability,Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants With Developmental and Epileptic Encephalopathies Followed by Open-Label Extension(OLE)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: SCN2A Encephalopathy
SCN8A Encephalopathy
Keywords: Pediatric epilepsy
NaV1.2 Voltage-Gated Sodium Channel
SCN2A variant
SCN8A variant
Developmental and epileptic encephalopathy
Lead sponsor: Praxis Precision Medicines
Sponsor class: INDUSTRY
First posted: 2023-04-19
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Parallel group
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* 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.
* Has a seizure frequency as follows:

  * At least 8 countable motor seizures in the 4 weeks immediately prior to Screening as reported by the parent/legal guardian or in the opinion of the investigator AND
  * At least 8 countable motor seizures during the 28 day Baseline Observation Period (during which seizure frequency is recorded in a daily seizure diary).
* Additional inclusion criteria apply and will be assessed by the study team.

Exclusion Criteria:

* Has any clinically significant or known pathogenic or likely pathogenic genetic variant other than in SCN2A and SCN8A or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder.
* Has a documented, functionally characterized loss-of-function (LoF) missense variant or a presumed LoF variant (nonsense or frameshift variant) based on genetic testing and/or clinical evidence that prior exposure to a sodium channel blocker (SCB) medication worsened seizures.
* Has 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening.
* Additional exclusion criteria apply and will be assessed by the study team.

Primary outcomes:
measure: To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs
timeFrame: 16 weeks
Secondary outcomes:
measure: To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs
timeFrame: 48 weeks
measure: Incidence of Treatment-Emergent Adverse Events (TEAEs) [Safety and Tolerability])
timeFrame: 16 weeks
measure: Plasma concentrations of PRAX-562
timeFrame: 16 weeks
measure: Seizure Frequency (OLE Extension)
timeFrame: 48 weeks
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Praxis Research Site
state: Georgia
city: Chicago
country: United States
facility: Praxis Research Site
state: Illinois
city: Minneapolis
country: United States
facility: Praxis Research Site
state: Minnesota
city: Hackensack
country: United States
facility: Praxis Research Site
state: New Jersey
city: Madrid
country: Spain
facility: Praxis Research Site
Study officials:
affiliation: Praxis Precision Medicines
name: Medical Director
role: STUDY_DIRECTOR
References: