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

November 8, 2023 updated by: Stoke Therapeutics, Inc

An Open-Label Extension Study for Patients With Dravet Syndrome Who Previously Participated in Studies of STK-001

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed 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.

Study Type

Interventional

Enrollment (Estimated)

69

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • California
      • San Francisco, California, United States, 94158
        • University of California San Francisco Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital
      • Orlando, Florida, United States, 32803
        • Florida Hospital for Children
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Children's Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Michigan Medicine
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • New York
      • New York, New York, United States, 10016
        • NYU Comprehensive Epilepsy Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health & Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • UT LeBonheur Pediatric Specialists, Inc.
    • Texas
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
      • Tacoma, Washington, United States, 98405
        • MultiCare Health System Institute for Research and Innovation

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Completed dosing with STK-001 and the End of Study Visit in Study STK-001-DS-101, with an acceptable safety profile per Investigator judgment.
  • Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 per Investigator and Sponsor judgment.
  • Completed Study STK-001-DS-101 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.
  • Currently treated with an antiepileptic drug (AED) acting primarily as a sodium channel blocker, as maintenance therapy, including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
  • 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.
  • Participating in an observational study, they are excluded unless approved by the Sponsor.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: STK-001 multiple dose levels
Enrollment of patients after completion of study STK-001-DS-101 if eligible for additional dosing in this extension study. Patients will receive IT administration of study drug STK-001 at the dose level they received while participating in Study STK-001-DS-101, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an STK-001 Phase 1/2 study, and doses above 30 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56.
STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of multiple doses of STK-001
Time Frame: Screening (Day -1) until 6 months after multiple drug dosing
Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and Gillette FAQ parameters.
Screening (Day -1) until 6 months after multiple drug dosing

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetic (PK) Parameters
Time Frame: Dosing (Day 1) until 6 months after multiple drug dosing
Analysis of plasma concentrations of STK-001
Dosing (Day 1) until 6 months after multiple drug dosing
Exposure of STK-001 in Cerebrospinal Fluid (CSF)
Time Frame: Dosing (Day 1) until Week 32 (last study drug dosing day)
Measurement of STK-001 concentrations
Dosing (Day 1) until Week 32 (last study drug dosing day)
Measurement of Seizure Frequency
Time Frame: Screening (Day -1) until 6 months after multiple drug dosing
Measurement of Seizure Frequency (by paper diary)
Screening (Day -1) until 6 months after multiple drug dosing
Change in overall clinical status
Time Frame: Screening (Day -1) until 6 months after multiple drug dosing
Change in overall clinical status as measured by the Clinical Global Impression of Change (CGIC) and the Caregiver Global Impression of Change (CaGIC)
Screening (Day -1) until 6 months after multiple drug dosing
Change in Quality of Life
Time Frame: Screening (Day -1) until 6 months after multiple drug dosing
Change in quality of life as measured by the EuroQoL-five dimensions, youth version (EQ-5D-Y) instrument
Screening (Day -1) until 6 months after multiple drug dosing

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Ann Dandurand, MD, Medical Director

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 21, 2021

Primary Completion (Estimated)

February 3, 2026

Study Completion (Estimated)

March 3, 2027

Study Registration Dates

First Submitted

February 2, 2021

First Submitted That Met QC Criteria

February 2, 2021

First Posted (Actual)

February 5, 2021

Study Record Updates

Last Update Posted (Actual)

November 9, 2023

Last Update Submitted That Met QC Criteria

November 8, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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