Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PRAX-562 · 1 trial · 2 indications
Changes from baseline in monthly (28-day) motor seizure frequency
Incidence and severity of TEAEs
| Arm | Type | Description |
|---|---|---|
| Part A: Randomized, Double-Blind 0.5mg/kg/day PRAX-562 or PRAX-562/Placebo | EXPERIMENTAL | 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). |
| Part B: Open-Label Extension Treatment 0.5mg/kg/day PRAX-562 | EXPERIMENTAL | Eligible participants will receive 0.5mg/kg/day administered orally or via G-tube for up to 144 weeks. |
| Part A: Randomized, Double-Blind 1.0 mg/kg/day PRAX-562 or PRAX-562/Placebo | EXPERIMENTAL | 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). |
| Open-Label Extension Treatment 1.0 mg/kg/day PRAX-562 | EXPERIMENTAL | Eligible participants will receive 1.0 mg/kg/day administered orally or via G-tube for up to 144 weeks. |
| Name | Type | Description |
|---|---|---|
| PRAX-562 | DRUG | Once daily oral or G-tube treatment. |
Inclusion Criteria: * Has a documented variant in SCN2A with onset of seizures occurring in the first 3 months of life or has a diagnosis of SCN8A-DEE supported by both clinical and genetic findings. * Has a seizure frequency as follows: * At least 8 countable motor seizures in the 4 weeks immed...
PRAX-562 is an investigational small molecule being developed for the treatment of SCN2A Encephalopathy, a developmental and epileptic encephalopathy. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
PRAX-562 targets the NaV sodium channel, a class of voltage-gated sodium channels involved in neuronal excitability. By modulating this target, the drug aims to address the underlying pathophysiology of SCN2A Encephalopathy and related developmental and epileptic encephalopathies.
PRAX-562 is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol PRAX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with developmental and epileptic encephalopathies.
PRAX-562 is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 2 trial is active but not recruiting participants, and the drug remains in clinical testing.
PRAX-562 is being evaluated in a single Phase 2 clinical trial with the identifier NCT05818553. This trial is a randomized, double-blind, controlled study enrolling approximately 77 participants with SCN2A Encephalopathy or SCN8A Encephalopathy. The trial is active but not recruiting and is being conducted in the United States, Israel, Spain, and the United Kingdom.
PRAX-562 is a unique investigational compound developed by Praxis Precision Medicines. No alternative names for this drug have been disclosed in the available clinical trial information. It is distinct from other sodium channel modulators in development.