An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
Launched by STOKE THERAPEUTICS, INC · Feb 2, 2021
Trial Information
Current as of June 26, 2025
Enrolling by invitation
Keywords
ClinConnect Summary
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 aci...
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Completed dosing with STK-001 and the End of Study Visit in Study STK-001-DS-101 or Study STK-001-DS-102, with an acceptable safety profile per Investigator judgment.
- • Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 or Study STK-001-DS-102 per Investigator and Sponsor judgment.
- • Completed Study STK-001-DS-101 or STK-001-DS-102 within 4 weeks of the start of their participation in Study STK-001-DS-501 unless approved by sponsor.
- Exclusion Criteria:
- • Met any withdrawal criteria from Study STK-001-DS-101 or STK-001-DS-102.
- • Currently treated with an antiepileptic drug (AED) acting primarily as a sodium channel blocker, as maintenance therapy, including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide or cenobamate.
- • 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 or STK-001-DS-102.
- • Participating in an observational study, they are excluded unless approved by the Sponsor.
About Stoke Therapeutics, Inc
Stoke Therapeutics, Inc. is a biotechnology company dedicated to advancing transformative therapies for genetic diseases by harnessing the power of RNA modulation. With a focus on precision medicine, Stoke aims to develop innovative treatments that address the underlying causes of conditions caused by insufficient protein expression. Through its proprietary platform, the company is committed to delivering novel solutions that improve patient outcomes and enhance quality of life. Stoke Therapeutics is driven by a mission to unlock the potential of RNA science for patients and their families, fostering a future where genetic disorders can be effectively managed or cured.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Rochester, Minnesota, United States
Boston, Massachusetts, United States
Philadelphia, Pennsylvania, United States
Washington, District Of Columbia, United States
Aurora, Colorado, United States
Miami, Florida, United States
Fort Worth, Texas, United States
Seattle, Washington, United States
Portland, Oregon, United States
Chicago, Illinois, United States
Iowa City, Iowa, United States
New York, New York, United States
Orlando, Florida, United States
Tacoma, Washington, United States
Ann Arbor, Michigan, United States
San Francisco, California, United States
Boston, Massachusetts, United States
Memphis, Tennessee, United States
Patients applied
Trial Officials
Ann Dandurand, MD
Study Director
Medical Director
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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