Dravet Syndrome
Conditions
Interventions
Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of
Sponsors
Ann M Dandurand
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Patients must be >=2 and <18 years of age. -Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by: Onset, prior to 12 months of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures.No other known etiology causing clinical DS manifestations. -Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the sodium voltage-gated channel type 1 alpha subunit (SCN1A) gene. -Patient must experience the required number of major motor seizures during the 6-week Observation Period. Major motor seizure types included are Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic. -Patient must have used at least 2 prior interventions for seizures. These can include anti-seizure medications (ASMs), ketogenic diet and/or vagus nerve stimulation (VNS) with either lack of adequate seizure control or discontinued due to an AE(s). These interventions can be ongoing therapies. -Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis for any indication will be considered an ASM. -Patients' maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS) must have been stable (unless adjusted for weight) during the Baseline Period.
Exclusion criteria
Exclusion criteria: -Patient has documented variant in the SCN1A gene associated with gain-of-function -Patient is currently treated with a maintenance ASM acting primarily as a sodium channel blocker, including but not limited to phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate, given the mechanism of action of zorevunersen. -Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS. -Patient has emergence of a new seizure type or reemergence of a past seizure type (seizure types that last occurred more than 12 months before Screening Visit A) during the Baseline Period, or has more than 1 hospitalization for seizures during the Baseline Period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Measurement of Seizure Reduction at Week 28 | — |
Secondary
| Measure | Time frame |
|---|---|
| Measurement of Seizure Reduction at Week 52 Multi-component Vineland-3 Outcome Score at Week 52 (Measurement of change from baseline) Vineland-3 Subdomain Score at Week 52 (Measurement of change from baseline) | — |
Countries
Japan, United Kingdom, United States of America
Contacts
Public ContactjRCT Inquiry Receipt Center
IQVIA Services Japan G.K.
Outcome results
None listed