A Phase 2,Double-Blind,Randomized Clinical Trial to Explore the Safety,Tolerability,Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants With Developmental and Epileptic Encephalopathies Followed by Open-Label Extension(OLE)
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 18
Healthy Volunteers: f
View:
• 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.
Locations
United States
Georgia
Praxis Research Site
ACTIVE_NOT_RECRUITING
Atlanta
Illinois
Praxis Research Site
RECRUITING
Chicago
Minnesota
Praxis Research Site
RECRUITING
Minneapolis
New Jersey
Praxis Research Site
RECRUITING
Hackensack
Other Locations
Spain
Praxis Research Site
RECRUITING
Madrid
Contact Information
Primary
Head of Pharmacovigilance
clinicaltrials@praxismedicines.com
617-300-8460
Time Frame
Start Date:2023-08-02
Estimated Completion Date:2027-03-01
Participants
Target number of participants:100
Treatments
Experimental: Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo
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).
Experimental: Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562
Eligible participants will receive 0.5mg/kg/day administered orally or via G-tube for up to 144 weeks.
Experimental: Part A: Randomized, Double-Blind 1.0 mg/kg/day PRAX-562 or PRAX-562/Placebo
Eligible participants from each cohort will be randomized in a 1:1 ratio to either 1.0 milligrams/kilograms/day (mg/kg/day) PRAX-562 for 16 weeks (PRAX-562 arm) or 1.0 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).