Dravet syndrome (participants =2.5 years of age) Nervous System Diseases
Conditions
Interventions
Current intervention as of 01/08/2023:
Participants who have completed the ADMIRAL (STK-001-DS-102) study will have the possibility of rolling over on to the Longwing (STK-001-DS-502) extension study.
Sponsors
Stoke Therapeutics, Inc
Eligibility
Sex/Gender
All
Age
2.5 Years to 100 Years
Inclusion criteria
Inclusion criteria: 1. Completed dosing with STK-001 and the End of Study Visit in Study STK 001-DS-102, with an acceptable safety profile per the Investigator's judgment 2. Satisfactory compliance with study visits and procedures in Study STK 001-DS-102 per Investigator and Sponsor judgment 3. Completed Study STK-001-DS-102 within 4 weeks of the start of their participation in Study STK-001-DS-502, unless approved by the Sponsor
Exclusion criteria
Exclusion criteria: 1. Met any withdrawal criteria from Study STK-001-DS-102 2. Current treatment as maintenance therapy with an antiepileptic drug acting primarily as a sodium channel blocker including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide 3. Clinically significant unstable medical conditions other than epilepsy 4. Clinically relevant symptoms or a clinically significant illness (in the judgment of the Investigator) in the 4 weeks prior to Screening/Baseline of Study STK-001-DS-502, other than epilepsy 5. Spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt 6. Prior treatment (or is being treated) with an investigational product (other than STK-001) since participating in Study STK 001 DS-102 7. Participating in an observational study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measures as of 01/08/2023: 1. Safety and tolerability of multiple doses of STK-001 from screening (day-1) until 6 months after multiple drug dosing, based on: 1.1. Incidence, type, severity, and seriousness of adverse events (AEs) measured by review of all reported events for all participants 1.2. Incidence of abnormal vital signs (including body temperature, heart rate, systolic and diastolic blood pressure, and respiratory rate) measured using standard methods such as thermometer and blood pressure cuff 1.3. Incidence of abnormal 12-lead electrocardiogram (ECG) findings (including PR, QRS, and QT intervals) measured using 12-lead electrocardiograms 1.4. Incidence of abnormal laboratory parameters (including haematology, coagulation, clinical chemistry and urine tests) measured using validated assays of blood and urine samples 1.5. Incidence of immunogenicity (anti-drug antibodies) measured using a validated assay in serum 1.6. Percent change from baseline in locomotor skill ability assessed using the Gillette Functional Assessment Questionnaire (FAQ) total score _____ Previous primary outcome measures: 1. Safety and tolerability of multiple doses of STK-001 from screening (day-1) until 6 months after multiple drug dosing, based on: 1.1. Incidence, type, severity, and seriousness of adverse events (AEs) measured by review of all reported events for all participants 1.2. Incidence of abnormal vital signs (including body temperature, heart rate, systolic and diastolic blood pressure, and respiratory rate) measured using standard methods such as thermometer and blood pressure cuff 1.3. Incidence of abnormal 12-lead electrocardiogram (ECG) findings (including PR, QRS, and QT intervals) measured using 12-lead electrocardiograms 1.4. Incidence of abnormal laboratory parameters (including haematology, coagulation, clinical chemistry and urine tests) measured using validated assays of blood and urine samples 1.5. Incidence of immunogenicity | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Pharmacokinetic (PK) parameters measured by analysis of plasma concentrations of STK-001 using hybridization ELISA from day 1 (dosing) until 6 months after multiple drug dosing 2. Exposure of STK-001 in cerebrospinal fluid (CSF) by measurement of STK-001 concentrations using hybridization ELISA from day 1 (dosing) until the last study drug dosing day 3. Seizure frequency measured using a paper diary from screening (day -1) until 6 months after multiple drug dosing 4. Overall clinical status as measured by the Clinician-assessed Global Impression of Change Scale from baseline (day -1) until 6 months after multiple drug dosing 5. Quality-of-life measured by EuroQoL-five dimensions, youth version (EQ-5D-Y) from baseline (day -1) until 6 months after multiple drug dosing | — |
Countries
England, Scotland, United Kingdom
Contacts
Public ContactStoke Therapeutics General Mailbox
Outcome results
None listed