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

  • STATUS
    Recruiting
  • End date
    Oct 8, 2022
  • participants needed
    48
  • sponsor
    Stoke Therapeutics, Inc
Updated on 5 November 2020
Investigator
Javier Avendaño, MD
Primary Contact
Children's National Medical Center (5.3 mi away) Contact
+11 other location
fever
epilepsy
seizure
tonic
ketogenic diet
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
Treatment STK-001
Clinical Study IdentifierNCT04442295
SponsorStoke Therapeutics, Inc
Last Modified on5 November 2020

Adding a note
adding personal notes guide

Select a piece of text and start making personal notes.

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 2 yrs and 18 yrs?
Gender: Male or Female
Do you have any of these conditions: Myoclonic Epilepsy or Dravet Syndrome?
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

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar
Name

Primary Contact

site
Name

Phone Email

0/250
Please verify that you are not a bot.
Step 2 Get screened

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

Learn more
Step 3 Enroll in the clinical study

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
Step 4 Get your study results

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

Loading...

Browse trials for

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

Study

user name

Annotated by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No made yet