What side effects are associated with this type of intervention?

Common treatments for Dravet Syndrome include antiepileptic drugs, genetic therapies, and molecular therapies. Genetic or molecular therapies, such as those similar to STK-001, often aim to modify the underlying genetic causes of the disorder. Known side effects of these treatments can include injection site reactions, immune responses, and potential off-target effects that may impact other genes or cellular functions. Additionally, there may be risks of infection, inflammation, and other systemic effects depending on the delivery method and the specific therapy used.

What prior FDA approvals exist?

The FDA has approved several treatments for Dravet Syndrome, including antiepileptic drugs like stiripentol, clobazam, and cannabidiol (CBD). These treatments primarily focus on managing seizures rather than addressing the genetic root cause of the syndrome. STK-001 represents a novel approach as it is a genetic therapy aimed at addressing the underlying genetic mutations in Dravet Syndrome. This type of molecular therapy is still under investigation, and no similar genetic therapies have been approved by the FDA for Dravet Syndrome as of now.

What other types of research exist?

Promising novel alternatives to STK-001 for Dravet Syndrome patients include: 1) Antisense oligonucleotide therapies targeting SCN1A mutations, which aim to restore normal function of the sodium channels affected in Dravet Syndrome. 2) Gene therapy approaches that deliver functional copies of the SCN1A gene to compensate for the defective gene. 3) CRISPR/Cas9-based gene editing techniques that correct the underlying genetic mutations in the SCN1A gene. These therapies are in various stages of research and clinical trials and represent potential future treatment options for Dravet Syndrome.

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Antisense Oligonucleotide

STK-001 for Dravet Syndrome

Phase 1 & 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

Use of at least 2 prior treatments for epilepsy that either had lack of adequate seizure control (requiring an additional AED) or had to be discontinued due to an AE(s)

Diagnosis of Dravet Syndrome (DS) with onset of recurrent focal motor or hemiconvulsive or generalized tonic-clonic seizures prior to 12 months of age, which are often prolonged and triggered by hyperthermia

Must not have

Clinically significant unstable medical conditions other than epilepsy

History of brain or spinal cord disease (other than epilepsy or DS), or history of bacterial meningitis or brain malformation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (day -1) until 6 months after single and multiple drug dosing

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing STK-001, a new medicine for people with Dravet syndrome. It aims to increase a protein in the brain that these patients lack, which could help reduce seizures and improve their overall health.

Who is the study for?

This trial is for children and adolescents with Dravet Syndrome, who had normal development at seizure onset and have been on stable epilepsy treatments for at least 4 weeks. They must have tried at least two other epilepsy treatments without success or stopped due to side effects. Participants should not have any significant medical conditions besides epilepsy.

What is being tested?

The study by Stoke Therapeutics tests the safety of different doses of STK-001 given once or multiple times in patients with Dravet syndrome. It's an open-label study, meaning everyone knows what treatment they're getting, and it also looks at how seizures and quality of life might change.

What are the potential side effects?

While specific side effects are not listed, the trial will monitor how safe STK-001 is when given either as a single dose or multiple doses over time. Side effects could range from mild to severe depending on individual reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have tried at least 2 epilepsy treatments that didn't work or caused side effects.

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I was diagnosed with Dravet Syndrome and had seizures before I was 1 year old.

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I have a genetic variation in the SCN1A gene linked to Dravet Syndrome.

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My tests show a specific genetic change in the SCN1A gene related to my condition.

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I've tried at least 2 epilepsy treatments that didn't work well or caused side effects.

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 have never had brain or spinal cord diseases, bacterial meningitis, or brain malformations, except for epilepsy or DS.

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I have a genetic mutation known to cause epilepsy.

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

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (day -1) until 6 months after single and multiple drug dosing

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (day -1) until 6 months after single and multiple drug dosing

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day -1) until 6 months after single and 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

Exposure of STK-001 in Cerebrospinal Fluid (CSF)

Pharmacokinetic (PK) Parameters

Safety and Tolerability of single and multiple doses of STK-001 with respect to

Secondary study objectives

Change in Caregiver Global Impression of Change Scale

Change in Clinician-assessed Global Impression of Change Scale

Measurement of Quality of Life

+1 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Single Ascending DosesExperimental Treatment1 Intervention

Enrollment of patients in two age groups. A Sentinel group of 2 patients aged 13 to 18 years of age, inclusive, and an expanded group of 2 patients 2 to 12 years of age to receive single doses. There will be an option to dose up to 6 additional patients at each dose level and an option to expand the maximum tolerated dose level with 5 additional patients.

Group II: Multiple Ascending DosesExperimental Treatment1 Intervention

Enrollment of patients in two age groups. A Sentinel group of 2 patients aged 13 to 18 years of age, inclusive, and an expanded group of 2 patients 2 to 12 years of age to receive multiple doses. There will be an option to dose up to 6 additional patients at each dose level and an option to expand the maximum tolerated dose level with 10 additional patients.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

STK-001 - Single Ascending Doses

2020

Completed Phase 2

~70

STK-001 - Multiple Ascending Doses

2020

Completed Phase 2

~70

0 / 10Eligibility 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 is a severe form of epilepsy often caused by mutations in the SCN1A gene, which encodes a sodium channel critical for neuronal function. Treatments like STK-001 aim to address the underlying genetic cause by increasing the expression of the healthy SCN1A gene, potentially reducing seizure frequency and severity. Other common treatments include valproate and clobazam, which enhance GABAergic inhibition to stabilize neuronal activity, and the ketogenic diet, which alters brain metabolism to reduce excitability. These treatments are crucial for managing symptoms and improving the quality of life for Dravet Syndrome patients, as they target both the genetic and symptomatic aspects of the disorder.