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

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Dravet Syndrome
Related Access Program
M. Scott Perry – Dravet Syndrome M. Scott Perry – Dravet Syndrome University of Colorado, Denver – Dravet Syndrome Children’s Hospital Medical Center, Cincinnati – Dravet Syndrome
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Brief Title

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

Official Title

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

Brief 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.

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 Phase

Phase 2

Study Type

Interventional

Primary Outcome

Safety of multiple doses of STK-001

Secondary Outcome

 Pharmacokinetic (PK) Parameters

Condition

Dravet Syndrome

Intervention

STK-001

Study Arms / Comparison Groups

 STK-001 multiple dose levels
Description:  Enrollment of patients after completion of study STK-001-DS-101 if eligible for additional dosing in this extension study. Patients will be administered the same dose level they received in study STK-001- DS-101 and will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses 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 5

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

69

Start Date

January 21, 2021

Completion Date

March 3, 2027

Primary Completion Date

February 3, 2026

Eligibility Criteria

        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.

Gender

All

Ages

30 Months - N/A

Accepts Healthy Volunteers

No

Contacts

Javier Avendaño, MD, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT04740476

Organization ID

STK-001-DS-501

Responsible Party

Sponsor

Study Sponsor

Stoke Therapeutics, Inc

Study Sponsor

Javier Avendaño, MD, Study Director, Medical Director

Verification Date

February 2022