What is the mechanism of action of this treatment?

Dravet Syndrome treatments traditionally include antiepileptic drugs like valproate and clobazam, which reduce seizure frequency by modulating neurotransmitter activity. However, genetic therapies like STK-001 aim to address the root cause by targeting the SCN1A gene mutations responsible for the disorder. These therapies work by correcting the defective gene or modulating its expression to restore normal neuronal function. This approach offers the potential for more effective and long-lasting seizure control, significantly improving the quality of life for Dravet Syndrome patients.

What side effects are associated with this type of intervention?

Common treatments for Dravet Syndrome include antiepileptic drugs (AEDs) like valproate, clobazam, and stiripentol, as well as newer therapies such as cannabidiol (CBD) and fenfluramine. Side effects of these treatments can vary: valproate may cause liver toxicity and weight gain; clobazam can lead to sedation and behavioral changes; stiripentol may result in loss of appetite and neutropenia; CBD is associated with diarrhea and elevated liver enzymes; and fenfluramine can cause decreased appetite and fatigue. Genetic or molecular therapies like STK-001 are still under investigation, but potential side effects could include immune reactions and off-target effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Dravet Syndrome, including stiripentol, cannabidiol (Epidiolex), and fenfluramine (Fintepla), which focus on reducing seizure frequency. STK-001, currently under study, represents a novel genetic therapy targeting the SCN1A gene mutations that cause Dravet Syndrome, aiming to address the condition at its genetic root.

What other types of research exist?

Promising novel alternatives to STK-001 for Dravet Syndrome patients include antisense oligonucleotide therapies like those targeting the SCN1A gene mutations, gene therapy approaches aiming to correct or compensate for the defective gene, and other molecular therapies that modulate the expression or function of the affected ion channels. These therapies are in various stages of research and clinical trials, showing potential for improved outcomes in managing Dravet Syndrome.

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Antisense Oligonucleotide

STK-001 for Dravet Syndrome

Phase 2

Waitlist Available

Research Sponsored by Stoke Therapeutics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must not have

Clinically significant unstable medical conditions other than epilepsy.

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up screening (day -1) until 6 months after multiple drug dosing

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety of STK-001, a new treatment for Dravet syndrome. It aims to help patients by increasing a brain protein that is usually low in this condition. The study focuses on patients who have already tried this treatment in earlier studies.

Who is the study for?

This trial is for patients with Dravet syndrome who completed the STK-001 study STK-001-DS-101, showed a good safety profile, and were compliant with that study's procedures. They shouldn't be on certain antiepileptic drugs like sodium channel blockers or have unstable medical conditions besides epilepsy.

What is being tested?

The trial tests the long-term safety of repeated doses of STK-001 in those previously treated for Dravet syndrome. It's an open-label extension where changes in seizure frequency, overall health status, and quality of life are also monitored.

What are the potential side effects?

Specific side effects aren't listed here but generally include any adverse reactions observed during previous treatments with STK-001. The focus is on long-term tolerability and any new side effects that might emerge from extended use.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any unstable health conditions except for epilepsy.

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I am currently on medication for epilepsy that includes drugs like phenytoin or carbamazepine.

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I have a spinal condition or a CSF drainage shunt that might affect spinal fluid flow.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ screening (day -1) until 6 months after multiple drug dosing

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~screening (day -1) until 6 months after multiple drug dosing

This trial's timeline: 3 weeks for screening, Varies for treatment, and screening (day -1) until 6 months after multiple drug dosing for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety of multiple doses of STK-001

Secondary study objectives

Change in Quality of Life

Change in overall clinical status

Exposure of STK-001 in Cerebrospinal Fluid (CSF)

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: STK-001 multiple dose levelsExperimental Treatment1 Intervention

Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. 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 STK-001-DS-102, 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 45 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.

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Dravet Syndrome treatments traditionally include antiepileptic drugs like valproate and clobazam, which reduce seizure frequency by modulating neurotransmitter activity. However, genetic therapies like STK-001 aim to address the root cause by targeting the SCN1A gene mutations responsible for the disorder. These therapies work by correcting the defective gene or modulating its expression to restore normal neuronal function. This approach offers the potential for more effective and long-lasting seizure control, significantly improving the quality of life for Dravet Syndrome patients.