PARTICIPATE IN RESEARCH

Dravet Syndrome Foundation feels strongly that research is our best hope for advancing treatments and eventually finding a cure for Dravet syndrome and associated epilepsies. Research is not just the responsibility of scientists and doctors. It is important that patients participate to whatever extent they are able.

Patients can participate in research in several ways, including: 1) sharing their information with scientists through patient registries so researchers can understand how Dravet syndrome affects individuals; and 2) volunteering for clinical studies or trials to help doctors understand how treatments affect patients. In rare diseases such as Dravet syndrome, it can be difficult for potential treatments to reach the required number of participants to be granted FDA approval. In the absence of large numbers, we must leverage the resources we do have, which include our strong patient community.

Clinical studies seek to answer questions such as:

  • Does this investigational drug work?
  • Does it work better than another medicine already available?
  • Does it cause any side effects?
  • Are there any other benefits that could improve patient quality of life?

CURRENT RESEARCH STUDIES IN DRAVET SYNDROME

The MONARCH Study, conducted by Stoke Therapeutics, is currently enrolling for a Phase1/2a trial of STK-001. This new therapeutic medicine acts to specifically increase the number of healthy sodium channels (Nav1.1) that are impacted by mutations in SCN1A gene, the most common cause of Dravet syndrome. The study will focus on safety and tolerability, as well as assessing the efficacy of STK-001 to reduce seizure activity and patient health. It should take 7-9 months to complete, including 1 dose of the medication, a total of 8 visits to the study site, and 3 phone calls.

Clinical trial information can be found on ClinicalTrials.gov.

A quick information sheet can be found here.

For more detailed information, please visit this link or email MONARCH@iqvia.com.

STK-001 is part of a new technology called TANGO that Stoke Therapeutics is leveraging to treat haploinsufficiencies like Dravet syndrome. If you would like to find out more about the science behind this approach, you can read this TANGO explainer provided by Stoke Therapeutics.

Epygenix is partnering with Greenlight Clinical to run a Phase 2 Clinical Trial for EPX-100 (Clemizole HCl) for children ages 2-17 years diagnosed with Dravet syndrome.  This is a multi-center, randomized, double-blind, placebo-controlled, proof-of-concept trial to evaluate the efficacy of EPX-100. The study will last for 20 weeks (4 week observation/ 4 week titration/ 12 week maintenance), with the option to enter a follow-up 52-week Open-Label Extension phase after the initial maintenance period. This 20-week study of EPX-100 versus placebo will primarily focus on seizure activity and related outcomes, but the study will also include assessments of quality of life and behavior.

This trial is not yet recruiting, but we will update as they begin actively recruiting patients.

Details of this trial are posted at ClinicalTrials.gov.

If you want to read more about the treatments Epgenix Therapeutics is developing, visit their website here.

DETAILS COMING SOON!

Encoded Therapeutics is currently finalizing plans for a Natural History Study in patients with Dravet syndrome.

In the meantime, if you’d like to know more about ETX-101, the viral gene therapy Encoded is developing for the treatment of Dravet syndrome, you can read more at their website.