Actively Recruiting
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
Led by Praxis Precision Medicines · Updated on 2025-08-21
60
Participants Needed
3
Research Sites
108 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying PRAX-222 in children aged 2 to 18 years who have early onset SCN2A developmental and epileptic encephalopathy (DEE), a rare form of epilepsy that begins before 3 months of age. This trial aims to learn about the safety and effects of multiple doses of PRAX-222 in this pediatric population. The study is sponsored by Praxis Precision Medicines and includes phases to assess safety and efficacy. Participants will receive different doses of PRAX-222 or a placebo in a randomized, double-blind design. The study includes an initial dose escalation phase with ascending doses, optional further dose escalations, fixed-dose treatment phases, and open-label treatment periods where all participants may receive PRAX-222. Placebo procedures are included to compare responses. The dosing schedule varies across these treatment phases. Throughout the trial, children will be monitored for treatment-emergent adverse events and seizure frequency at various timepoints up to 92 weeks. Assessments include seizure counts, clinical global impression scores, developmental milestones, EEG measures, behavior scales, quality of life, and sleep evaluations. This comprehensive monitoring helps researchers understand the treatment's safety and impact. Participation may last up to nearly two years depending on study phases.
CONDITIONS
Brief Title
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Has onset of seizures prior to 3 months of age
- Has a minimum weight of at least 10 kg at screening
- Has a documented SCN2A variant through accredited genetic testing
- Additional inclusion criteria apply and will be assessed by the study team
You will not qualify if you...
- Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder
- Is taking more than 2 sodium channel blocking anti-seizure medications
- Additional exclusion criteria apply and will be assessed by the study team
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 22 weeks
Participants receive initial and ascending doses of PRAX-222 or placebo to assess safety and dosing.
Multiple visits over the course of dosing
Duration - Variable, overlapping with Dose Escalation Phase
Participants may receive optional ascending doses of PRAX-222 or placebo for further dose escalation.
Visits as required for dose escalation
Duration - Approximately 4 to 8 weeks
Participants receive double-blind fixed doses of PRAX-222 or placebo to confirm efficacy and safety.
Regular visits during confirmatory dosing phase
Duration - Up to 92 weeks
Participants receive open-label PRAX-222 after blinded phases to continue treatment.
Visits scheduled according to treatment protocol
Trial Site Locations
Total: 3 locations
1
Le Bonheur Childrens Hospital
Memphis, Tennessee, United States, 38103
Completed
2
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90035-003
Actively Recruiting
3
Praxis Research Site
São Paulo, Brazil, 05403-010
Actively Recruiting
Research Team
H
Head of Pharmacovigilance
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
8
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