Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05737784 | A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy | PHASE1 | RECRUITING | 60 | — | — | Apr 13, 2023 | Sep 1, 2027 | Aug 21, 2025 | 3 | United States, Brazil |
The number of participants with treatment-emergent adverse events will be reported by severity and preferred term.
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.
| Arm | Type | Description |
|---|---|---|
| Preliminary Safety | EXPERIMENTAL | Open-label PRAX-222 |
| Dose Escalation - PRAX-222 | EXPERIMENTAL | Initial dose escalation consisting of double-blind ascending doses of PRAX-222 |
| Dose Escalation - Placebo | PLACEBO_COMPARATOR | Double-blind placebo procedure |
| Optional Dose Escalation - PRAX-222 | EXPERIMENTAL | Optional dose escalation consisting of double-blind ascending doses of PRAX-222 |
| Optional Dose Escalation - Placebo | PLACEBO_COMPARATOR | Double-blind placebo procedure |
| Confirmatory Dosing - PRAX-222 | EXPERIMENTAL | Double-blind fixed-dose PRAX-222 |
| Confirmatory Dosing - Placebo | EXPERIMENTAL | Double-blind placebo procedure |
| Open-label PRAX-222 | EXPERIMENTAL | Open-label PRAX-222 |
| Name | Type | Description |
|---|---|---|
| PRAX-222 - Initial Dose | DRUG | PRAX-222 |
| PRAX-222 - Initial Ascending Doses | DRUG | Ascending doses of PRAX-222 |
| PRAX-222 - Optional Ascending Doses | DRUG | Escalation of PRAX-222 dose(s) |
| PRAX-222 - Fixed Doses | DRUG | Fixed-dose(s) of PRAX-222 not to exceed the maximum tolerated dose of PRAX-222 |
| Placebo | PROCEDURE | Placebo procedure |
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 Pat...