An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Dravet Disease
  • Age: Between 2 - 18 Years
  • Gender: Male or Female
  • Other Inclusion Criteria:
    1. Participants and/or authorized representative must provide written informed consent
    2. Participants and their caregivers must be willing and able (in the Investigator’s opinion) to comply with all protocol requirements.
    3. Participants must be diagnosed with Dravet syndrome (DS):
      • Onset prior to 12 months of age with recurrent focal motor or hemiconvulsive orgeneralized tonic-clonic seizures, which are often prolonged and triggered by hyperthermia
      • No past history of causal magnetic resonance imaging (MRI) lesion (MRI not required to confirm absence of lesion)
      • No other known etiology
      • Normal development at seizure onset
    4. Must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene associated with DS.
    5. Participants had at least 2 prior treatments for epilepsy that either had lack of adequate seizure control or had to be discontinued
    6. Participants must be experiencing 4 or more convulsive seizures during the initial 28 days of the Observation Period.
    7. Must currently be taking at least one AED at a dose which has been stable for at least 4 weeks prior
    8. All epilepsy medications or interventions for epilepsy must have been stable for at least 4 weeks
    9. Any marijuana- or cannabinoid-based product or medication is allowed but treatment must have been stable for at least 4 weeks prior
    10. Must be sufficiently fluent in English to be able to complete questionnaires relevant to this study

You may not be eligible for this study if the following are true:

    1. Has a known pathogenic mutation in another gene that causes epilepsy
    2. Currently being treated with an AED acting primarily as a sodium channel blocker,as maintenance treatment including: phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide
    3. Clinically significant unstable medical conditions other than epilepsy
    4. Clinically relevant symptoms or a clinically significant illness in the 4 weeks other than epilepsy
    5. History of brain or spinal cord disease or a history of bacterial meningitis or brain malformation
    6. Has spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt
    7. Has a psychiatric or behavioral disorder
    8. Female participants of childbearing potential and male participants whose partners are of childbearing potential who will not take contraceptive methods during the study and for 3 months thereafter
    9. Pregnant, lactating, or planning pregnancy during the course of the study
    10. Has any other significant disease or disorder which, in the opinion of the Investigator, may either put the participant at risk because of participation in the study, may influence the results of the study, or may affect the participation in the study



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