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


About this study

The purpose of this study is to evaluate the safety and tolerability of single-ascending doses of STK-001 in patients with Dravet Syndrome.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • 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.
  • Patient and their caregiver must be willing and able (in the Investigator’s opinion) to comply with all protocol requirements.
  • Patient must be between 2 and 18 years (inclusive) of age at Screening.
  • Patient must have DS as defined by:
    • Onset prior to 12 months of age with recurrent focal motor or hemiconvulsive or generalized 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.
  • Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene. Patients who have SCN1A testing results of Negative (no variants of clinical significance identified) cannot be enrolled.
  • Patient has had at least 2 prior treatments for epilepsy that either had lack of adequate seizure control (requiring an additional antiepileptic drug [AED]) or had to be discontinued due to an AE(s).
  • Patient must be experiencing 4 or more convulsive seizures (Hemiclonic, Focal With Motor Signs, Focal To Bilateral Tonic Clonic Convulsion, Generalized Tonic Clonic Convulsion, Tonic, Tonic/Atonic [Drop Attacks], and Clonic) during the initial 28 days of the Observation Period.
  • Patient must currently be taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening.
  • All epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) must have been stable (including product type, dose, and setting) for at least 4 weeks prior to Screening.
  • Any marijuana- or cannabinoid-based product or medication is allowed but treatment must have been stable for at least 4 weeks prior to Screening, including supplier, ratio, and dose.
  • Patient must meet age-appropriate institutional guidelines for intrathecal (IT) drug administration procedures.
  • Patient and/or family (or caretaker) must be sufficiently fluent in English or local language to be able to complete questionnaires relevant to this study.

Exclusion Criteria:

  • Patient has one of the following mutations in the SCN1A gene:
    • Thr226Met, Leu263Val, Val422Leu, Thr1174Ser, Trp1204Arg, Pro1345Ser, Gln1489Lys, Phe1499Leu, Arg1575Cys, Val1611Phe, Leu1624Pro, Arg1648Cys, Leu1649Gln, Leu1670Trp, Gly1674Arg, or Asp1866Tyr.
  • Patient has a known pathogenic mutation in another gene that causes epilepsy. (The pathogenic mutation must be homozygous in cases of known recessive disease).
  • Patient is currently being treated with a sodium channel blocker as maintenance treatment including: phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
  • Patient has clinically significant unstable medical conditions other than epilepsy.
  • Patient has had clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Screening or prior to dosing on Day 1, other than epilepsy.
  • Patient has a history of brain or spinal cord disease (other than epilepsy or DS) or a history of bacterial meningitis or brain malformation.
  • Patient has a spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt.
  • Patient has clinically significant (in the judgment of the Investigator) abnormal laboratory values at Screening or prior to dosing on Day 1.
  • Patient has aspartate aminotransferase or alanine aminotransferase > 2.5-fold upper limit of normal (ULN), serum creatinine > ULN or platelet count

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Elaine Wirrell, M.D.

Contact us for the latest status

Contact information:

Bridget Neja C.N.A.

(507) 266-9150

More information


Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available


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