A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

Phase 2

Recruiting

• Conditions: SCN2A Encephalopathy; SCN8A Encephalopathy

• Registration Number: NCT05818553
• Lead Sponsor: Praxis Precision Medicines

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-03-17
• Study First Submitted QC: 2023-04-17
• Study First Posted: 2023-04-19
• Study Start: 2023-08-02
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-08
• Last Update Posted: 2024-01-10
• Primary Completion: 2024-06-30
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events (TAEs) [Safety and Tolerability])	16 weeks	The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs	48 weeks	Incidence and severity of TEAEs

Secondary Outcome Measures

Name	Time	Method
To assess the effect of PRAX-562 on the frequency of countable motor seizures in pediatric participants with DEEs	16 weeks	Changes from baseline in monthly (28-day) motor seizure frequency
Plasma concentrations of PRAX-562	16 weeks	Sparse pharmacokinetic (PK) sampling will be used to calculate mean concentrations at baseline, and at Weeks 2, 4, 6, 8,10, 12 and 16.
Seizure Frequency (OLE Extension)	48 weeks	Efficacy assessments (seizure diary) will be collected daily and reviewed at timepoints Day 1, Week 16, Week 32, and Week 48.

Trial Locations

Locations (1)

Praxis Research Site; 🇪🇸 Madrid, Spain