A Study to Evaluate Patients with Dravet Syndrome Who Previously Participated in Studies of STK-001


About this study

The purpose of this study is to gather additional information on the long-term safety, tolerability, and impact on seizure frequency and duration of repeated doses of STK-001 administered every 4 months by intrathecal (IT) injection.


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 must be ≥ 2.5 years of age.
  • 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 have completed dosing with STK-001 and the End of Study Visit in Study STK-001-DS-101, with an acceptable safety profile per Investigator judgment.
  • Patient must have satisfactory compliance with study visits and procedures in Study STK-001-DS-101 per Investigator and Sponsor judgment.
  • Patient must meet age-appropriate institutional standard practices for intrathecal (IT) drug administration procedures.
  • Patient and/or family (or caretaker) must be sufficiently fluent in English or Spanish to be able to complete questionnaires relevant to this study.
  • Patient must have completed Study STK-001-DS-101 within 4 weeks of the start of their participation in Study STK-001-DS-501, unless approved by the Sponsor.

Exclusion Criteria:

  • Patient has met any withdrawal criteria from Study STK-001-DS-101.
  • Patient is currently being treated as maintenance therapy with an antiepileptic drug acting primarily as a sodium channel blocker 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 judgment of the Investigator) at Screening or prior to dosing on Day 1, other than epilepsy.
  • 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 baseline or prior to dosing on Day 1.
  • Patient has aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3-fold upper limit of normal (ULN), serum creatinine > ULN or platelet count < lower limit of normal at baseline and upon repeat testing.
  • Patient has clinically significant abnormalities (in the judgment of the Investigator) in the 12-lead ECG measured at Screening/Baseline (This includes 12-lead ECG from STK-001-DS-101 [Visit 6 or Visit 9], if STK-001-DS-101 Visit 6 or Visit 9 and STK-001-DS-501 Visit 1 are the Same Day).
  • Patient has a psychiatric or behavioral disorder which, in the opinion of the Investigator, may interfere with the patient’s participation in the study.
  • Patient is currently taking, or within 4 weeks prior to Screening/Baseline has taken any anticoagulant (including but not limited to heparins, warfarin and other vitamin K antagonists, dabigatran, rivaroxaban and apixaban), or within 7 days prior to Screening/Baseline, has taken any antiplatelet (including but not limited to aspirin, non-steroidal anti-inflammatory drugs, clopidogrel, ticlopidine. and dipyridamole).
  • Patient is a female of childbearing potential, or patient is a fertile male with female partner(s) of childbearing potential, unless willing to ensure that they or their partners use effective contraception throughout the duration of the study and for at least 6 months after their last dose of STK-001.
  • . Patient is a female who is lactating or planning pregnancy during the duration of the study and for at least 6 months after their last dose of STK-001.
  • Patient has any other significant disease or disorder which, in the opinion of the Investigator, may either put the patient at risk because of participation in the study, may influence the results of the study, or may affect the patient’s ability to participate in the study.
  • Patient has been treated (or is being treated) with an investigational product (other than STK-001) since participating in Study STK-001-DS-101.
  • Patient is participating in an observational study, unless approved by the Sponsor.

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.

Closed-enrolling by invitation

What is this? (?)
Not open to everyone who meets the eligibility criteria, but only those invited to participate by the study team.

Contact information:

Bridget Neja C.N.A.

(507) 266-9150


More information


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