Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as PRAX-222 - Initial Dose, PRAX-222- Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222- Initial Ascending Doses, PRAX-222 - Optional Ascending Doses, PRAX-222- Optional Ascending Doses, PRAX-222 - Fixed Doses, PRAX-222- Fixed Doses
Elsunersen · 2 trials · 4 indications
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
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 |
|---|---|---|
| Cohort 1 Arm 1: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 2: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| Cohort 3: Open-Label Treatment Period | EXPERIMENTAL | Open-label elsunersen |
| 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 |
|---|---|---|
| 1mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| 0.5mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| 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 a documented Gain of Function SCN2A variant confirmed through genetic testing. * Has onset of seizures prior to 3 months of age. * Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period. Exclusion Criteria: * Has any cli...
Elsunersen is an investigational small molecule being developed for epileptic encephalopathy, specifically SCN2A encephalopathy, a form of developmental and epileptic encephalopathy. It is currently in Phase 3 clinical development and has not been approved by the FDA.
Elsunersen targets the SCN2A gene, which encodes a sodium channel subunit. As an antisense oligonucleotide (ASO), it is designed to modulate SCN2A expression. It is being studied in patients with SCN2A-related epileptic encephalopathy.
Elsunersen is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on NASDAQ under the ticker PRAX. The company is conducting a Phase 3 clinical trial of elsunersen in pediatric patients with SCN2A developmental and epileptic encephalopathy.
Elsunersen is currently in Phase 3 clinical development. It is an investigational drug and has not received FDA approval. The drug has been granted Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations by the FDA for its intended indication.
Elsunersen is being evaluated in a Phase 3 clinical trial with the identifier NCT07019922. This trial is recruiting an estimated 40 participants across the United States, Brazil, Germany, and Italy. The study is open to pediatric patients as young as 1 day old with SCN2A encephalopathy.
Yes, elsunersen is also known as 1mg elsunersen. This alternative name may be used in certain contexts, but both refer to the same investigational drug being developed by Praxis Precision Medicines for SCN2A-related epileptic encephalopathy.