An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Dravet Syndrome
About this trial
This is an interventional treatment trial for Dravet Syndrome focused on measuring Pediatric epilepsy, Epileptic Encephalopathies, Refractory Myoclonic Epilepsy, Severe Myoclonic Epilepsy in Infancy
Eligibility Criteria
Inclusion Criteria:
- 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.
- Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 per Investigator and Sponsor judgment.
- Completed Study STK-001-DS-101 within 4 weeks of the start of their participation in Study STK-001-DS-501 unless approved by sponsor.
Exclusion Criteria:
- Met any withdrawal criteria from Study STK-001-DS-101.
- Currently treated with an antiepileptic drug (AED) acting primarily as a sodium channel blocker, as maintenance therapy, including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
- Clinically significant unstable medical conditions other than epilepsy.
- 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.
- Spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt.
- Treated (or is being treated) with an investigational product (other than STK-001) since participating in Study STK-001-DS-101.
- Participating in an observational study, they are excluded unless approved by the Sponsor.
Sites / Locations
- University of California San Francisco Medical Center
- Children's Hospital Colorado
- Children's National Medical Center
- Nicklaus Children's Hospital
- Florida Hospital for Children
- Ann & Robert H. Lurie Children's Hospital of Chicago
- University of Iowa Children's Hospital
- Massachusetts General Hospital
- Michigan Medicine
- Mayo Clinic
- NYU Comprehensive Epilepsy Center
- Oregon Health & Science University
- Children's Hospital of Philadelphia
- UT LeBonheur Pediatric Specialists, Inc.
- Cook Children's Medical Center
- Seattle Children's Hospital
- MultiCare Health System Institute for Research and Innovation
Arms of the Study
Arm 1
Experimental
STK-001 multiple dose levels
Enrollment of patients after completion of study STK-001-DS-101 if eligible for additional dosing in this extension study. Patients will receive IT administration of study drug STK-001 at the dose level they received while participating in Study STK-001-DS-101, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an STK-001 Phase 1/2 study, and doses above 30 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56.