A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Purpose
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
Eligibility
- Eligible Ages
- Between 2 Years and 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
Exclusion Criteria
- 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
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- 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.
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
- Masking Description
- No masking in the 2 open-label parts, masking as indicated in the 2 double-blind, randomized parts.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
More Details
- NCT ID
- NCT05737784
- Status
- Recruiting
- Sponsor
- Praxis Precision Medicines