An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Last updated: May 7, 2024
Sponsor: Stoke Therapeutics, Inc
Overall Status: Active - Enrolling

Phase

2

Condition

Dravet Syndrome

Unverricht-lundborg Syndrome

Seizure Disorders (Pediatric)

Treatment

STK-001

Clinical Study ID

NCT04740476
STK-001-DS-501
  • Ages > 30
  • All Genders

Study Summary

Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Completed dosing with STK-001 and the End of Study Visit in Study STK-001-DS-101 orStudy 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-101or 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 theirparticipation in Study STK-001-DS-501 unless approved by sponsor.

Exclusion

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 sodiumchannel 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 ofthe 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 animplanted 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 theSponsor.

Study Design

Total Participants: 60
Treatment Group(s): 1
Primary Treatment: STK-001
Phase: 2
Study Start date:
January 21, 2021
Estimated Completion Date:
March 03, 2027

Study Description

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.

Connect with a study center

  • University of California San Francisco Medical Center

    San Francisco, California 94158
    United States

    Site Not Available

  • Children's Hospital Colorado

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Children's National Medical Center

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Nicklaus Children's Hospital

    Miami, Florida 33155
    United States

    Site Not Available

  • Florida Hospital for Children

    Orlando, Florida 32803
    United States

    Site Not Available

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois 60611
    United States

    Site Not Available

  • University of Iowa Children's Hospital

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Michigan Medicine

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • NYU Comprehensive Epilepsy Center

    New York, New York 10016
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Site Not Available

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • UT LeBonheur Pediatric Specialists, Inc.

    Memphis, Tennessee 38103
    United States

    Site Not Available

  • Cook Children's Medical Center

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Seattle Children's Hospital

    Seattle, Washington 98105
    United States

    Site Not Available

  • MultiCare Health System Institute for Research and Innovation

    Tacoma, Washington 98405
    United States

    Site Not Available

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