Dravet syndrome is a medication-resistant developmental and epileptic encephalopathy that usually begins in the first year of life and accumulates significant lifelong symptom burden. It is associated with frequent seizures and multiple neurologic and systemic comorbidities. Many patients carry pathogenic variants in SCN1A.
New treatments for rare pediatric neurological conditions show promise, with zorevunersen reducing seizures in Dravet syndrome patients and an SMA therapy under FDA review following stem cell research breakthroughs.
Stoke Therapeutics presented long-term Dravet syndrome data showing up to 80% seizure reductions sustained through three years, outlined its Phase 3 EMPEROR trial design targeting mid-2027 data, and disclosed first patient dosing in its ADOA program.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07592650 |
Applicability and Effectiveness of Remote Occupational Therapy |
COMPLETED | |
| NCT07531745 |
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome |
RECRUITING | PHASE1/PHASE2 |
| NCT07251673 |
Longitudinal Study of Phenotypic and Developmental Severity in Patients With Dravet Syndrome With SCN1A Gene Mutation |
RECRUITING | |
| NCT07176832 |
Bioavailability of Stiripentol After Single Oral Dose of Capsule vs Suspension in Healthy Subjects (STILIQ) |
COMPLETED | PHASE1 |
| NCT07112365 |
The FINTEPLA as an Anti-SUDEP Therapy in Dravet Syndrome Project |
NOT_YET_RECRUITING | PHASE4 |
| NCT07013331 |
A PET-MRI Study of Serotoninergic Brainstem Pathway in Patients With Dravet Syndrome |
RECRUITING | NA |
| NCT06872125 |
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome |
RECRUITING | PHASE3 |
| NCT06660394 |
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS) |
RECRUITING | PHASE3 |
| NCT06504511 |
SCN1A Horizons A Natural History Study of SCN1A-related Epilepsies in the United Kingdom |
RECRUITING | |
| NCT06401538 |
BMB-101 in Absence Epilepsy and DEE |
RECRUITING | PHASE2 |
