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
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug, Procedure
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 18
Healthy Volunteers: f
View:
• 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 genetic testing obtained via a laboratory accredited per Clinical Laboratory Improvement Amendments (CLIA) or College of American Pathologists (CAP) or equivalent.
• Additional inclusion criteria apply and will be assessed by the study team
Locations
United States
Tennessee
Le Bonheur Childrens Hospital
COMPLETED
Memphis
Other Locations
Brazil
Hospital de Clinicas de Porto Alegre
RECRUITING
Porto Alegre
Praxis Research Site
RECRUITING
São Paulo
Contact Information
Primary
Head of Pharmacovigilance
clinicaltrials@praxismedicines.com
617-300-8460
Time Frame
Start Date: 2023-04-13
Estimated Completion Date: 2027-09
Participants
Target number of participants: 60
Treatments
Experimental: Preliminary Safety
Open-label PRAX-222
Experimental: Dose Escalation - PRAX-222
Initial dose escalation consisting of double-blind ascending doses of PRAX-222
Placebo_comparator: Dose Escalation - Placebo
Double-blind placebo procedure
Experimental: Optional Dose Escalation - PRAX-222
Optional dose escalation consisting of double-blind ascending doses of PRAX-222
Placebo_comparator: Optional Dose Escalation - Placebo
Double-blind placebo procedure
Experimental: Confirmatory Dosing - PRAX-222
Double-blind fixed-dose PRAX-222
Experimental: Confirmatory Dosing - Placebo
Double-blind placebo procedure
Experimental: Open-label PRAX-222
Open-label PRAX-222
Related Therapeutic Areas
Sponsors
Leads: Praxis Precision Medicines