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A Multi-Center, Single-Arm Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 1mg elsunersen | Drug | 24 weeks every 4 weeks intrathecally |
|
| Measure | Description | Time Frame |
|---|---|---|
| To assess the efficacy of elsunersen on seizure frequency in participants with early-onset SCN2A DEE | Median percent change in monthly (28 days) motor seizure frequency from baseline to treatment after 24 weeks | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE | Responder rate - defined as a ≥50% reduction in monthly seizure frequency from baseline compared to treatment after 24 weeks | 24 weeks |
| To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Head of Pharmacovigilance | Contact | 617-300-8460 | clinicaltrials@praxismedicines.com |
| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Praxis Precision Medicines | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Praxis Research Site | Recruiting | San Diego | California | 92123 | United States | |
| Praxis Research Site |
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Specific milestones have to be met in Cohort 1 for enrollment to open in Cohort 2 and the same will apply to open Cohort 3.
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| 0.5mg elsunersen | Drug | 24 weeks every 4 weeks intrathecally |
|
Change in motor seizure-free days from baseline |
| 24 weeks |
| CGI-S change from baseline | CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this patient population and rate the current severity on a 7-point scale from 1 (normal, not at all ill) to 7 (among the most extremely ill patients). | 24 weeks |
| CGI subdomain scores at each postdose time point | Clinical Global Impression-Improvement (CGI-I) subdomains scores at each postdose time point | 24 weeks |
| CgGI-S from baseline | Caregiver Global Impression-Severity (CgGI-S) at baseline compared to treatment after 24 weeks | 24 weeks |
| CgGI-I subdomain scores at each postdose time point | Caregiver Global Impression-Improvement (CgGI I) subdomains scores at each postdose time point | 24 weeks |
| Sleep assessment scores from baseline | Sleep assessment scores at baseline compared to each postdose time point | 24 weeks |
| To evaluate the safety and tolerability of elsunersen in participants with early-onset SCN2A DEE | Incidence and severity of treatment-emergent adverse events (TEAEs) | 24 weeks |
| Recruiting |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Praxis Research Site | Recruiting | Chicago | Illinois | 60612 | United States |
| Praxis Research Site | Recruiting | Cincinnati | Ohio | 45229 | United States |
| Praxis Research Site | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Praxis Research Site | Recruiting | Memphis | Tennessee | 38105 | United States |
| Praxis Research Site | Withdrawn | Porto Alegre | 90035 | Brazil |
| Praxis Research Site | Withdrawn | São Paulo | 05403 | Brazil |
| Praxis Research Site | Recruiting | Bonn | Bonn | 53127 | Germany |
| Praxis Research Site | Recruiting | Rome | 00165 | Italy |
| ID | Term |
|---|---|
| D004827 | Epilepsy |
| D012640 | Seizures |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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