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Studies

Interventional Active not recruiting Phase 1/Phase 2

NCT05737784 on ClinicalTrials.gov

A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

Official title: A Seamless, Clinical Trial to Investigate the Safety and Efficacy of Multiple Doses of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

  • Sponsor: Praxis Precision Medicines
  • Enrollment: 60 (Estimated)
  • Study start: 2023-04-13 (Actual)
  • Primary completion: 2026-04-01 (Actual)
  • Study completion: 2028-02-01 (Estimated)
  • Allocation: Randomized
  • InterventionModel: Parallel
  • InterventionModelDescription: Seamless study design with sequential study parts, including an open-label safety part; a double-blind, randomized dose-escalation part; a double-blind, randomized confirmatory part; and an open-label extension part.
  • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • PrimaryPurpose: Treatment

Brief Summary

The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.

Conditions

  • SCN2A-DEE
  • Epilepsy

Intervention / Treatment

  • Drug: PRAX-222 - Initial Dose
  • Drug: PRAX-222 - Initial Ascending Doses
  • Drug: PRAX-222 - Optional Ascending Doses
  • Drug: PRAX-222 - Fixed Doses
  • Procedure: Placebo

Primary Outcome Measures

  • Number of participants with treatment-emergent adverse events (Preliminary Safety, Dose Escalation) (Screening (-8 weeks) through up to 92 weeks)
    The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
  • Seizure frequency (Confirmatory Phase) (36 to 40 weeks)
    Seizure frequency will be captured by a seizure diary and outcomes will be measured by summing the seizure frequency over a 28-day time period following the 6th dose administration in the confirmatory phase.

Countries

Brazil, United States

Locations and Investigators

  • Le Bonheur Childrens Hospital, Memphis, United States
  • Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil
  • Praxis Research Site, São Paulo, Brazil

Study Officials

  • Medical Director (Study Director) — Praxis Precision Medicines