Brief Title
An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Official Title
An Open-Label Extension Study for Patients With Dravet Syndrome Who Previously Participated in Studies of STK-001
Brief 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.
Detailed Description
This study is a multi-center, open-label, multiple-dose, safety extension study for patients who have completed another study of STK-001 and meet study eligibility criteria. STK-001 is an investigational new medicine for the treatment of Dravet syndrome. STK-001 is an antisense oligonucleotide (ASO) that is intended to increase the level of productive SCN1A messenger RNA (mRNA) and consequently increase the expression of the sodium channel Nav1.1 protein. This RNA-based approach is not gene therapy, but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA). STK-001 is designed to upregulate Nav1.1 protein expression from the nonmutant (wild-type) copy of the SCN1A gene to restore physiological Nav1.1 levels. Nav1.1 levels are reduced in people with Dravet syndrome. Stoke has generated preclinical data demonstrating proof-of-mechanism for STK-001.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Safety of multiple doses of STK-001
Secondary Outcome
Pharmacokinetic (PK) Parameters
Condition
Dravet Syndrome
Intervention
STK-001
Study Arms / Comparison Groups
STK-001 multiple dose levels
Description: Enrollment of patients after completion of study STK-001-DS-101 if eligible for additional dosing in this extension study. Patients will be administered the same dose level they received in study STK-001- DS-101 and will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses 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 5
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
69
Start Date
January 21, 2021
Completion Date
March 3, 2027
Primary Completion Date
February 3, 2026
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.
Gender
All
Ages
30 Months - N/A
Accepts Healthy Volunteers
No
Contacts
Javier Avendaño, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04740476
Organization ID
STK-001-DS-501
Responsible Party
Sponsor
Study Sponsor
Stoke Therapeutics, Inc
Study Sponsor
Javier Avendaño, MD, Study Director, Medical Director
Verification Date
February 2022