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Also known as 1mg elsunersen
elsunersen · 1 trial · 2 indications
Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks
| 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 |
| Name | Type | Description |
|---|---|---|
| 1mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
| 0.5mg elsunersen | DRUG | 24 weeks every 4 weeks intrathecally |
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 developmental and epileptic encephalopathy (DEE). It is a -sen class antisense oligonucleotide (ASO) therapy. The drug is currently in Phase 3 clinical development and has not been approved by the FDA.
Elsunersen is an antisense oligonucleotide (ASO), a class of therapy that targets RNA to modulate gene expression. It is being studied in patients with SCN2A encephalopathy, a genetic form of epileptic encephalopathy. The drug is designed to address the underlying genetic cause of this condition.
Elsunersen is being developed by Praxis Precision Medicines, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol 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 been approved by the FDA. The drug has received Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations from the FDA for its intended use in epileptic encephalopathy.
Elsunersen is being evaluated in a Phase 3 clinical trial registered as NCT07019922, titled 'A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety.' The trial is currently recruiting participants in the United States, Brazil, Germany, and Italy, with an enrollment target of 40 patients.
Yes, elsunersen is also known as 1mg elsunersen. This alternative name may be used in some contexts to refer to the same investigational drug. The drug is being developed by Praxis Precision Medicines for the treatment of SCN2A developmental and epileptic encephalopathy.