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

  • STATUS
    Recruiting
  • participants needed
    48
  • sponsor
    Stoke Therapeutics, Inc
Send
Updated on 5 August 2020
See if I qualify
fever
epilepsy
tonic
seizure
ketogenic diet
dravet syndrome
scn1a gene

Summary

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

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.

Details
Condition Myoclonic Epilepsy, dravet syndrome
Age 2years - 18years
Treatment STK-001
Clinical Study IdentifierNCT04442295
SponsorStoke Therapeutics, Inc
Last Modified on5 August 2020

Eligibility

 Yes No Not Sure

Inclusion Criteria

Must have DS with onset prior to 12 months of age with recurrent focal motor or hemiconvulsive or generalized tonic-clonic seizures, 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
Had at least 2 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 be taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening
All epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) must have been stable (including product type, dose, and setting) for at least 4 weeks prior to Screening

Exclusion Criteria

Known pathogenic mutation in another gene that causes epilepsy
Currently being treated with an antiepileptic drug acting primarily as a sodium channel blocker including: phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide
Clinically significant unstable medical conditions other than epilepsy
Has had 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
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
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
Message sent successfully.
We have submitted the information you provided to the research team at the location you chose. For your records, we have sent a copy of the message to your email address.
If you would like to be informed of other studies that may be of interest to you, you may sign up for Patient Notification Service.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Browse trials for

fever
epilepsy
tonic
seizure
ketogenic diet
dravet syndrome
scn1a gene

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.