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

P

Praxis Precision Medicines

Status and phase

Enrolling
Phase 2

Conditions

SCN8A Encephalopathy
SCN2A Encephalopathy

Treatments

Drug: Part A: 0.5mg/kg/day PRAX-562 for 12 Weeks and Matching Placebo for 4 Weeks
Drug: Part B: 0.5mg/kg/day PRAX-562
Drug: Part A: 0.5mg/kg/day PRAX-562 for 16 Weeks

Study type

Interventional

Funder types

Industry

Identifiers

NCT05818553
PRAX-562-221

Details and patient eligibility

About

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.

Enrollment

20 estimated patients

Sex

All

Ages

2 to 18 years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Triple Blind

20 participants in 2 patient groups

Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo
Experimental group
Description:
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).
Treatment:
Drug: Part A: 0.5mg/kg/day PRAX-562 for 16 Weeks
Drug: Part A: 0.5mg/kg/day PRAX-562 for 12 Weeks and Matching Placebo for 4 Weeks
Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562
Experimental group
Description:
Eligible participants will receive 0.5mg/kg/day administered orally or via G-tube for 48 weeks.
Treatment:
Drug: Part B: 0.5mg/kg/day PRAX-562

Trial contacts and locations

0

Loading...

Central trial contact

Head of Pharmacovigilance

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location

Resources

© Copyright 2024 Veeva Systems