Stoke Therapeutics STK-001-DS-502 - Longwing (extension study)
Research type
Research Study
Full title
Longwing: An Open-Label Extension Study for Patients with Dravet Syndrome who Previously Participated in Studies of STK-001
IRAS ID
1004380
Contact name
Shamim Ruff
Contact email
Sponsor organisation
Stoke Therapeutics, Inc
Eudract number
2021-005626-14
Clinicaltrials.gov Identifier
Research summary
Dravet syndrome (DS) is a very severe and difficult to treat epilepsy presenting in the first year of life with seizures and later significant learning disability with poor long-term prognosis. The primary goal of therapy for DS is to reduce seizure frequency and severity. There remains a significant need for additional therapies for patients to address the other DS comorbidities.
DS is caused by a mutation in the SCN1A gene leading to loss of function resulting in reduced levels of the protein. The study drug, is intended to increase the expression of the protein using an RNA based approach.
This extension study is open for children and teenagers who completed the main study (ADMIRAL). During the ADMIRAL study, participants received one of the doses of STK-001 that was being looked at. In this study, participants will receive the same dose of STK-001 as they received in the ADMIRAL study or a dose recommended by the medical experts overseeing the safety of the study.
The main purpose of this extension study is to look at how safe the STK-001 is when given every 4 months and for a longer period. The study will also look at how well a person can handle receiving multiple doses of STK-001 (called “tolerability”).
In addition, the study will look at:
• how multiple doses of the study drug are taken up and broken down by the body (called “pharmacokinetics”)
• the effect of multiple doses of the STK-001 on preventing seizures
• the effect of multiple doses of the STK-001 on the overall symptoms and comorbidities of DS and on the quality of life in children and adolescents with DS.
Up to 60 participants between age 2.5-18 years are expected to enrol in this study. Duration of the study for participants is up to 15 months/or for patients tolerating treatment, study duration will continue with treatment every 4 months (16 weeks) until the end of the study, unacceptable toxicity, withdrawal of consent, or Investigator/Sponsor decision, whichever comes first.REC name
Scotland A: Adults with Incapacity only
REC reference
22/SS/0005
Date of REC Opinion
26 Apr 2022
REC opinion
Further Information Favourable Opinion