An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

• Status: Enrolling by invitation
• Trial end date: 2024-09-01
• Target enrollment: 0
• Participant gender: All

Summary

Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Stoke Therapeutics, Inc

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.

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.