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. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints.
Patients who participated in the MONARCH study and meet study entry criteria are eligible to continue treatment in SWALLOWTAIL, an open label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001.
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
Downloadable Resources
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. Stoke also intends to measure non-seizure aspects of the disease, such as overall clinical status and quality of life, as secondary endpoints.
Patients who participated in the ADMIRAL study and meet study entry criteria are eligible to continue treatment in LONGWING, an open label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001.
Autosomal Dominant Optic Atrophy
FALCON Natural History Study
FALCON is a multicenter, prospective natural history study of people ages 8 to 60 who have an established clinical diagnosis of ADOA that is caused by a heterozygous OPA1 gene variant. No investigational medications or other treatments will be provided. The study is expected to enroll patients across 10 sites in the U.S., U.K., Italy and Denmark.
Trial Status
Currently enrolling
Location
U.S., U.K., Italy and Denmark
CONTACT FOR PARTICIPATION
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.
