Clinical Studies

Current and planned clinical studies

Click to expand the relevant category to learn more about our current and planned clinical studies. For more information about study participation you can also email us at clinicaltrials@stoketherapeutics.com.

Dravet Syndrome

Monarch Phase 1/2a

MONARCH is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study are to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective is to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints. Stoke plans to enroll approximately 48 patients in the study across 20 sites in the United States.

Trial Status

Currently recruiting

Location

United States

Website

Visit MONARCHstudy.com

Butterfly Observational Study

BUTTERFLY is an observational study of Dravet syndrome patients initiated by Stoke. This non-interventional two-year study, being conducted at approximately 20 sites in the United States, observes children and adolescents ages 2 to 18 years with mutations in the SCN1A gene.

Trial Status

Enrollment complete

Location

United States

Contact For Participation

BUTTERFLYStudy@iqvia.com

Downloadable Resources

BUTTERFLY Study Fact Sheet

Admiral Phase 1/2a

The ADMIRAL study is a Phase 1/2a open-label study of children and adolescents ages 2 to <18 who have an established diagnosis of Dravet syndrome and have evidence of a genetic mutation in the SCN1A gene. The primary objectives for the study are to assess the safety and tolerability of multiple doses of STK-001, as well as to characterize human pharmacokinetics. A secondary objective is to assess the effect of multiple doses of STK-001 as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 24-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as overall clinical status and quality of life, as secondary endpoints. Stoke plans to enroll up to 60 children and adolescents in the study across multiple sites in the United Kingdom.

Trial Status

Currently recruiting

Location

United Kingdom

Website

Visit admiralstudy.com

Autosomal Dominant Optic Atrophy

Natural history data collection

Will initiate in the second half of 2021.

Please return to this page for updates.

Trial Status

Coming soon

Location

Pending

Epilepsy Gene Panel Testing Program

As the potential for precision therapies in epilepsy becomes clear, genetic testing for patients is critical. Stoke Therapeutics is part of an innovative, cross-company collaboration founded by Invitae and BioMarin that aims to provide faster diagnosis for young children with epilepsy. The collaboration sponsors a no-cost epilepsy gene panel testing program for health care providers (HCPs) to order for any child up to the age of 8 years old who has had an unprovoked seizure.

“Stoke science is leading us toward solutions that we believe will make a real difference for patients and their families. We are committed to working with a sense of urgency while we conduct robust clinical trials that are designed to provide the necessary evidence to gain approval.”

– Nancy M. Wyant, SVP, Development Operations

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