Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PRAX-222 - Initial Dose
PRAX-222- Initial Dose · 1 trial · 2 indications
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...
PRAX-222 is an investigational small molecule being developed for SCN2A developmental and epileptic encephalopathy (SCN2A-DEE), a severe early-onset epilepsy syndrome. It is currently in Phase 1 clinical development and has not been approved by the FDA.
PRAX-222 is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol PRAX. The company is conducting clinical trials of PRAX-222 in pediatric patients with SCN2A-DEE.
PRAX-222 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 trial is currently active but not recruiting participants.
PRAX-222 is being studied in a Phase 1 clinical trial with the identifier NCT05737784. This trial is a randomized, double-blind, placebo-controlled study in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy, enrolling approximately 60 patients in the United States and Brazil.
Yes, PRAX-222 is also known as PRAX-222 - Initial Dose. Both names refer to the same investigational drug being developed by Praxis Precision Medicines for the treatment of SCN2A-DEE.