Dravet SyndromePhase 1/2NCT04442295

An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

A Phase 1/2 study testing STK-001 - Single Ascending Doses and STK-001 - Multiple Ascending Doses in people with Dravet Syndrome, run by Stoke Therapeutics, Inc at 18 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency, overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study. Read more about this study

Who is it for?: Ages 2 to 18 years
Conditions the study is for: Dravet Syndrome
What is being tested?: STK-001 - Single Ascending Doses and STK-001 - Multiple Ascending Doses
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 62 participants
Where: 18 sitesUnited States
Study dates: 29 Jun 2020to 14 Nov 2023
Phase: Phase 1/2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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.
No history of causal MRI lesion
No other known etiology
Normal development at seizure onset.
Documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene associated with DS.
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).
Currently taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening.
Stable epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) for at least 4 weeks prior to Screening.

Who cannot join

Known pathogenic mutation in another gene that causes epilepsy
Currently treated with an AED acting primarily as a sodium channel blocker, as maintenance treatment, 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 4 weeks prior to Screening or prior to dosing on Day 1, other than epilepsy.
History of brain or spinal cord disease (other than epilepsy or DS), or history of bacterial meningitis or brain malformation
Spinal deformity or other condition that may alter the free flow of cerebrospinal fluid (CSF) or has an implanted CSF drainage shunt.
Any other significant disease or disorder which, in the opinion of the Investigator, may either put the patient at risk because of participation in the study, may influence the results of the study, or may affect the patient's ability to participate in the study.

Trial locations and contacts

18 sites in 1 country.

City, postcode or hospital

18 sites in 1 country

United States18 sites

University of Michigan - Mott Children's Hospital
Ann Arbor, Michigan, 48109
Children's Hospital Colorado
Aurora, Colorado, 80045
Massachusetts General Hospital - Pediatric Epilepsy Program
Boston, Massachusetts, 02114
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cook Children's Health Care System
Fort Worth, Texas, 76104
University of Iowa Hospitals and Clinics; Pediatric Specialty Clinic
Iowa City, Iowa, 52242
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105
Nicklaus Children's Hospital
Miami, Florida, 33155
NYU Comprehensive Epilepsy Center
New York, New York, 10016
AdventHealth Orlando
Orlando, Florida, 32803
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55905
Primary Children's Hospital
Salt Lake City, Utah, 84108
UCSF Benioff Children's Hospital
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Multicare Institute for Research and Innovation
Tacoma, Washington, 98405
Children's National Medical Center
Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents With Dravet Syndrome

Read the detailed description

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.

Conditions: Dravet Syndrome
Being tested: STK-001 - Single Ascending Doses, STK-001 - Multiple Ascending Doses
Keywords: Pediatric epilepsy, Epileptic Encephalopathies, Refractory Myoclonic Epilepsy, Severe Myoclonic Epilepsy in Infancy
Medical subject headings: Epilepsies, Myoclonic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

STK-001 - Single Ascending DosesDrug
Experimental : Single Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Four dose levels will be evaluated ( 10mg, 20mg,30mg, 45mg and 70mg ).
STK-001 - Multiple Ascending DosesDrug
Experimental : Multiple Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Three dose levels will be evaluated ( 20mg,30mg and 45mg ).

Study groups in NCT04442295
Study group	Role	What they receive
Single Ascending Doses	Experimental	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. Receives: Drug: STK-001 - Single Ascending Doses
Multiple Ascending Doses	Experimental	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. Receives: Drug: STK-001 - Multiple Ascending Doses

What is being measured

The primary outcome is the main question the study is designed to answer.

Safety and Tolerability of single and multiple doses of STK-001 with respect to:
1. Incidence of adverse events
2. incidence of abnormal vital signs
3. Abnormal physical examination findings
4. Abnormal 12-lead electrocardiogram (ECG)
5. Abnormal laboratory parameters
Measured: Screening (Day -28) until 6 months after single and multiple drug dosing
Pharmacokinetic (PK) Parameters
Analysis of plasma concentrations of STK-001
Measured: Day 1 (Dosing) until 6 months after single and multiple drug dosing
Exposure of STK-001 in Cerebrospinal Fluid (CSF)
Measurement of STK-001 concentrations
Measured: Day 1 (Dosing) until 6 months after single and multiple drug dosing

Secondary outcomes (4)

Measurement of seizure frequency
Measured by paper diary
Measured: Screening (Day -28) until 6 months after single and multiple drug dosing
Change in Caregiver Global Impression of Change Scale
Change from baseline in overall clinical status as measured by the Clinical Global Impression of Change (CGIC).
Values of scales:
1. Very much improved
2. Much improved
3. Minimally improved
4. No change
5. Minimally worse
6. Much worse
7. Very much worse
Measured: Baseline (Day -1) until 6 months after single and multiple drug dosing
Change in Clinician-assessed Global Impression of Change Scale
Change from baseline in overall clinical status as measured by the Caregiver Global Impression of Change (CaGIC)
Values of scales:
1. Very much improved
2. Much improved
3. Minimally improved
4. No change
5. Minimally worse
6. Much worse
7. Very much worse
Measured: Baseline (Day -1) until 6 months after single and multiple drug dosing
Measurement of Quality of Life
Change in quality of life as measured by the EuroQoL-five dimensions, youth version (EQ-5D-Y) instrument. The scale is scored from 0-100. The reference to a high score indicates a better outcome of quality of life.
Measured: Baseline (Day -1) until 6 months after single and multiple drug dosing

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

29 Jun 202014 Nov 2023

First posted22 Jun 2020
Study start29 Jun 2020
Primary completion14 Nov 2023
Study completion14 Nov 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Dravet SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Enrolment: 62 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04442295
Sponsor's protocol ID: STK-001-DS-101
Status last verified: May 2025
First submitted: 8 Jun 2020
Last updated: 18 May 2025
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Laux L, Sullivan J, Perry MS, Brunklaus A, Desurkar A, Schreiber JM, Roberts CM, Knupp KG, Wheless JW, Wirrell EC, Ventola P, Wang F, Meena, Lynch J, Parkerson KA, Ticho B, Cross JH; MONARCH, ADMIRAL, SWALLOWTAIL, and LONGWING Study Groups. Zorevunersen in Children and Adolescents with Dravet Syndrome. N Engl J Med. 2026 Mar 5;394(10):969-982. doi: 10.1056/NEJMoa2506295.
PubMed 41780062

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Dravet Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 18 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents With Dravet Syndrome
ClinicalTrials.gov ID: NCT04442295
Acronym / sponsor ID: STK-001-DS-101
Conditions: Dravet Syndrome
Keywords: Pediatric epilepsy, Epileptic Encephalopathies, Refractory Myoclonic Epilepsy, Severe Myoclonic Epilepsy in Infancy
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 62 participants (actual)
Lead sponsor: Stoke Therapeutics, IncIndustry (pharma, biotech, devices)
Study start: 29 Jun 2020 (actual)
Primary completion: 14 Nov 2023 (actual)
Study completion: 14 Nov 2023 (actual)
First posted: 22 Jun 2020 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Sequential
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Single Ascending Doses	Experimental	Drug: STK-001 - Single Ascending DosesEnrollment 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.
Multiple Ascending Doses	Experimental	Drug: STK-001 - Multiple Ascending DosesEnrollment 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.

Who may qualify

Ages: 2 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (8 listed)

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.
Documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene associated with DS.
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).
Currently taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening.
Stable epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) for at least 4 weeks prior to Screening.

Key exclusion criteria (7 listed)

Known pathogenic mutation in another gene that causes epilepsy
Currently treated with an AED acting primarily as a sodium channel blocker, as maintenance treatment, 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 4 weeks prior to Screening or prior to dosing on Day 1, other than epilepsy.
History of brain or spinal cord disease (other than epilepsy or DS), or history of bacterial meningitis or brain malformation

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
STK-001 - Single Ascending DosesExperimental : Single Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Four dose levels will be evaluated ( 10mg, 20mg,30mg, 45mg and 70mg ).	Drug	Single Ascending Doses
STK-001 - Multiple Ascending DosesExperimental : Multiple Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Three dose levels will be evaluated ( 20mg,30mg and 45mg ).	Drug	Multiple Ascending Doses

Primary outcome

Safety and Tolerability of single and multiple doses of STK-001 with respect to:
Time frame: Screening (Day -28) until 6 months after single and multiple drug dosing
1. Incidence of adverse events 2. incidence of abnormal vital signs 3. Abnormal physical examination findings 4. Abnormal 12-lead electrocardiogram (ECG) 5. Abnormal laboratory parameters
Pharmacokinetic (PK) Parameters
Time frame: Day 1 (Dosing) until 6 months after single and multiple drug dosing
Analysis of plasma concentrations of STK-001
Exposure of STK-001 in Cerebrospinal Fluid (CSF)
Time frame: Day 1 (Dosing) until 6 months after single and multiple drug dosing
Measurement of STK-001 concentrations

4 secondary outcomes

Measurement of seizure frequency
Time frame: Screening (Day -28) until 6 months after single and multiple drug dosing
Change in Caregiver Global Impression of Change Scale
Time frame: Baseline (Day -1) until 6 months after single and multiple drug dosing
Change in Clinician-assessed Global Impression of Change Scale
Time frame: Baseline (Day -1) until 6 months after single and multiple drug dosing
Measurement of Quality of Life
Time frame: Baseline (Day -1) until 6 months after single and multiple drug dosing

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2025.
Derived from this study: PubMed 41780062Laux L, Sullivan J, Perry MS, Brunklaus A, Desurkar A, Schreiber JM, Roberts CM, Knupp KG, Wheless JW, Wirrell EC, Ventola P, Wang F, Meena, Lynch J, Parkerson KA, Ticho B, Cross JH; MONARCH, ADMIRAL, SWALLOWTAIL, and LONGWING Study Groups. Zorevunersen in Children and Adolescents with Dravet Syndrome. N Engl J Med. 2026 Mar 5;394(10):969-982. doi: 10.1056/NEJMoa2506295.

Related trials for Dravet Syndrome

Not yet recruitingPhase 3
Open-label, Single-arm Trial of Ganaxolone in CDD Patients 6 Months to Less Than 2 Years Old
CDKL5 Deficiency Disorder
RecruitingPhase 1/2
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Dravet Syndrome
RecruitingPhase 3
A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome
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Active, not recruitingPhase 3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Ann Dandurand, MD study director

Medical Director

18 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing STK-001 - Single Ascending Doses and STK-001 - Multiple Ascending Doses in people with Dravet Syndrome, run by Stoke Therapeutics, Inc at 18 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and Tolerability of single and multiple doses of STK-001 with respect to: (time frame: Screening (Day -28) until 6 months after single and multiple drug dosing). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04442295, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2025.