STK-001 for Dravet Syndrome

Recruiting at 16 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Stoke Therapeutics, Inc

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing the safety of STK-001, a new treatment for Dravet syndrome. It aims to help patients by increasing a brain protein that is usually low in this condition. The study focuses on patients who have already tried this treatment in earlier studies.

Research Team

Ann Dandurand, MD

Principal Investigator

Medical Director

Eligibility Criteria

This trial is for patients with Dravet syndrome who completed the STK-001 study STK-001-DS-101, showed a good safety profile, and were compliant with that study's procedures. They shouldn't be on certain antiepileptic drugs like sodium channel blockers or have unstable medical conditions besides epilepsy.

Inclusion Criteria

Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 per Investigator and Sponsor judgment.

I finished my STK-001 treatment and was deemed safe to proceed by my doctor.

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

I do not have any unstable health conditions except for epilepsy.

You are currently using or have used an experimental drug other than STK-001 after joining Study STK-001-DS-101.

I am currently on medication for epilepsy that includes drugs like phenytoin or carbamazepine.

Treatment Details

Interventions

• STK-001 (Antisense Oligonucleotide)

Trial Overview The trial tests the long-term safety of repeated doses of STK-001 in those previously treated for Dravet syndrome. It's an open-label extension where changes in seizure frequency, overall health status, and quality of life are also monitored.

Participant Groups

Group I: STK-001 multiple dose levels Experimental Treatment 1 Intervention

Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. 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 STK-001-DS-102, 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 45 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.

Who Is Running the Clinical Trial?

Stoke Therapeutics, Inc

Lead Sponsor

Trials

3

Recruited

270+