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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day -1) until 6 months after single and multiple drug dosing
Awards & highlights

Study Summary

This trial is testing a new medication for Dravet syndrome to see if it is safe and effective.

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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
Change in Caregiver Global Impression of Change Scale
Change in Clinician-assessed Global Impression of Change Scale
Measurement of Quality of Life
+1 more

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.

Find a Location

Who is running the clinical trial?

Stoke Therapeutics, IncLead Sponsor
1 Previous Clinical Trials
69 Total Patients Enrolled
Javier Avendaño, MDStudy DirectorMedical Director
1 Previous Clinical Trials
69 Total Patients Enrolled
Ann Dandurand, MDStudy DirectorMedical Director
6 Previous Clinical Trials
538 Total Patients Enrolled

Media Library

STK-001 (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04442295 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is eligibility for this trial restricted to individuals who are over twenty?

"This research requires that participants satisfy the age criteria of between 2 and 18 years old."

Answered by AI

Are there any opportunities to enroll in this research project at present?

"Confirmed, this clinical trial is currently in its recruitment phase. The initial posting was made on June 3rd 2020 and the most recent update occurred February 17th 2022."

Answered by AI

What are the aims of this research endeavor?

"According to the sponsor of this trial, Stoke Therapeutics Inc., it seeks to examine safety and tolerability over a period beginning on Day 1 (dosing) up until 6 months post-single and multiple drug administration. The primary outcome will be evaluated alongside secondary outcomes such as changes in caregiver global impression of change scales ranging from very much improved to very much worse; measurement of seizure frequency recorded by paper diary; and clinician-assessed global impression of change scale which is rated between very much improved or worsened."

Answered by AI

How many participants are currently undergoing the trial?

"Indeed, clinicaltrials.gov confirms that this trial is accepting new participants. It was first announced on June 3rd 2020 and the latest update came out on February 17th 2022. 78 individuals are needed across 24 medical centres for enrolment in this research project."

Answered by AI

Am I qualified to volunteer for this clinical experiment?

"A diagnosis of dravet syndrome (DS) and age between 2 to 18 are prerequisites for this study. Approximately 78 patients are sought after by the research team."

Answered by AI

How numerous are the locales currently conducting this experiment?

"24 medical sites are currently involved in this research project, with notable examples including Primary Children's Hospital located in Salt Lake City, Massachusetts General Hospital servicing Boston and the surrounding area, and Cook Children's Medical Centre situated near Fort Worth."

Answered by AI

Who else is applying?

What state do they live in?
Ohio
How old are they?
18 - 65
What site did they apply to?
University of Michigan - Mott Children's Hospital
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome
2020. "An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04442295.
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~6 spots leftby Sep 2024
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