An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
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|ClinicalTrials.gov Identifier: NCT04740476|
Recruitment Status : Enrolling by invitation
First Posted : February 5, 2021
Last Update Posted : February 10, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Dravet Syndrome||Drug: STK-001||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||69 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study for Patients With Dravet Syndrome Who Previously Participated in Studies of STK-001|
|Actual Study Start Date :||January 21, 2021|
|Estimated Primary Completion Date :||February 3, 2026|
|Estimated Study Completion Date :||March 3, 2027|
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.
STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection.
- Safety of multiple doses of STK-001 [ Time Frame: Screening (Day -1) until 6 months after multiple drug dosing ]Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and Gillette FAQ parameters.
- Pharmacokinetic (PK) Parameters [ Time Frame: Dosing (Day 1) until 6 months after multiple drug dosing ]Analysis of plasma concentrations of STK-001
- Exposure of STK-001 in Cerebrospinal Fluid (CSF) [ Time Frame: Dosing (Day 1) until Week 32 (last study drug dosing day) ]Measurement of STK-001 concentrations
- Measurement of Seizure Frequency [ Time Frame: Screening (Day -1) until 6 months after multiple drug dosing ]Measurement of Seizure Frequency (by paper diary)
- Change in overall clinical status [ Time Frame: Screening (Day -1) until 6 months after multiple drug dosing ]Change in overall clinical status as measured by the Clinical Global Impression of Change (CGIC) and the Caregiver Global Impression of Change (CaGIC)
- Change in Quality of Life [ Time Frame: Screening (Day -1) until 6 months after multiple drug dosing ]Change in quality of life as measured by the EuroQoL-five dimensions, youth version (EQ-5D-Y) instrument
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|Ages Eligible for Study:||30 Months and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04740476
|Study Director:||Javier Avendaño, MD||Medical Director|
|Responsible Party:||Stoke Therapeutics, Inc|
|Other Study ID Numbers:||
|First Posted:||February 5, 2021 Key Record Dates|
|Last Update Posted:||February 10, 2023|
|Last Verified:||February 2023|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
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