Padaczka: badanie fazy III leku STK-001

Kryteria włączenia kto może wziąć udział (9)

1. Patient and/or authorized representative must be willing and able to give informed consent/assent and any authorizations required by local law for participation in the study.
2. Patient and their caregiver must be willing and able (in the Investigator’s opinion) to comply with all protocol requirements.
3. At signing of consent/assent, patient must be ≥2 and <18 years of age.
4. Patient 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, which are often prolonged and triggered by hyperthermia. No past history of causal magnetic resonance imaging (MRI) lesion (past MRI or study-associated MRI not required to confirm absence of lesion). No other known etiology causing clinical DS manifestations. Normal development at seizure onset.
5. 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. Patients who have SCN1A testing results of Negative (no variants identified) cannot be randomized.
6. Experiences the required number of major motor seizures during the 6-week Observation Period. 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.
7. 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.
8. Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis (i.e., not as needed [PRN]) for any indication will be considered an ASM.
9. All maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS), as well as any marijuana- or cannabinoid-based products, must have been stable (unless adjusted for weight) during the Baseline Period. Felbamate must have been stable (unless adjusted for weight) for at least 6 months prior to Screening Visit A and during the Baseline Period. VNS implantation must have occurred at least 6 months prior to Screening Visit A. Rescue ASMs used on an as needed basis do not need to be stable.