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 : April 5, 2021
|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 :||48 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 :||September 2024|
|Estimated Study Completion Date :||September 2024|
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 be administered the same dose level they received in study STK-001-DS-101 and will receive a maximum of 3 doses. One dose will be administered every 4 months.
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 ]Analysis of incidence, type, severity, and seriousness of adverse events, vital signs, physical examination, electrocardiogram (ECG), and laboratory 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
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
|United States, California|
|University of California San Francisco Medical Center|
|San Francisco, California, United States, 94158|
|United States, Florida|
|Nicklaus Children's Hospital|
|Miami, Florida, United States, 33155|
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|Study Director:||Javier Avendaño, MD||Medical Director|