Participate in Research2018-03-28T13:15:18+00:00


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?


This study is open to patients 18-65 years of age, with Development and/or Epileptic Encephalopathy (DEE) have refractory seizures that are not resolved with the available treatment options and meet inclusion criteria. These seizures are often associated with cognitive, neurologic and behavioral problems. Development and/or Epileptic Encephalopathy can be caused by a broad range of etiologies including genetic and acquired, for example Lennox Gastaut, tuberous sclerosis, Dravet syndrome, stroke, hypoxic ischemic injury, etc.

Inclusion Criteria:

  1. Has a documented clinical diagnosis of developmental and/or epileptic encephalopathies with countable bilateral motor seizures, defined as an average of ≥2 per month during the past 3 months, based on the investigator’s assessment, and a monthly average of ≥1 per month during the Baseline Period, based on the seizure diary record.
  2. Has been taking 1 to 4 antiepileptic drug (AEDs) at a stable dose for ≥4 weeks before Screening and the participant or participant’s legally acceptable representative is willing to keep the regimen(s) stable throughout the study.
  3. Has an average of ≥1 bilateral motor seizure per month during the 4-week Baseline Period (i.e., drop seizures, tonic-clonic, tonic, bilateral clonic, atonic, myoclonic-atonic, myoclonic-tonic-clonic, focal seizures with bilateral hyperkinetic motor features).
  4. Must agree to not post any participant’s personal medical data related to the study or information related to the study on any web site or social media site (e.g., Facebook, Twitter) until the study has been completed.

The purpose of this study is to characterize the safety and tolerability of TAK-935, an investigational drug, in adult patients with developmental and/or epileptic encephalopathies. It will enroll approximately 20 patients.

This study has two parts:

Part 1: Participants will be randomly assigned (by chance, like flipping a coin) to one of the two treatment groups which will remain undisclosed to the patient and study doctor during the study (unless there is an urgent medical need). One group will receive TAK-935 100 mg, and the other group will receive placebo (dummy inactive pill) – this is a tablet that looks like the study drug but has no active ingredient.

Part 2: All participants who complete Part 1 will have the option to continue directly into the Open-Label treatment period in Part 2 where all patients will receive the study drug.

This study is conducted in 11 clinics around North America. The duration of this study is 121 days (approximately 4 months).

Please review the full summary of criteria at, at this link. If you believe you are eligible to participate, contact the closet site or the Takeda Study Registration Call Center at (877) 825-3327 or

ptc_logos-color2dPTC Therapeutics, the Dravet Syndrome Foundation and FACES are sponsoring a trial of Ataluren to treat children with treatment-resistant epilepsy associated with Dravet syndrome or CDKL5 due to stop codon mutations. These are DNA changes in which one DNA nucleotide is substituted for another one and instead of the three letter DNA sequence coding for an amino acid, it codes for a message to prematurely stop the amino acid chain and prematurely terminate the SCN1A or CDKL5 protein, resulting in a roughly 50% reduction in the amount of functional protein since these partial proteins are typically metabolized rapidly by nerve cells. Eight children with Dravet syndrome and eight children with CDKL5 will be enrolled.

Ataluren is a drug which reads through premature stop codons and increases the level of functional protein. The trial will begin in October 2016 at the NYU Langone Medical Center. Orrin Devinsky, MD, is the Principal Investigator. Eligibility for inclusion are children with documented premature stop codons in one of the allele for the SCN1A or CDKL5 genes. Children must be between ages 2 and 12 years and must have failed to achieve seizure control of 2 or more antiepileptic drugs and a current regimen of 1 to 3 baseline antiepileptic drugs. They must have a minimum of 6 convulsive or drop seizures with duration > 3 seconds over the 4 weeks of screening before study entry. The study visits and medication will be free of charge but all the visits are in NYC at the center so you will have travel back and forth every 6 weeks. The study should last approximately one year. During the study, patients will be continue their usual medications and be randomized to receive Ataluren during study period 1 or 2 and Placebo during the other study period. After the study is complete, all children will be given the opportunity to receive Ataluren. You can find criteria for study participation here, or please contact the study coordinator, Latoya King, if you have any further questions.

 zogenix-fb-pp-finalTWO (2) FAiRE Trial (Fenfluramine Assessment in Rare Epilepsy) in Children & Young Adults with Dravet syndrome  

Zogenix is conducting two (2) multicenter, double-blind, parallel-group, placebo-controlled, studies in North America to assess the efficacy, safety, and PK of ZX008 when used as adjunctive therapy for uncontrolled seizures in pediatric and young adult subjects with Dravet syndrome.





GWP CARE Clinical Trial Program

The GWP CARE clinical trial program is currently enrolling children with Dravet syndrome (DS) to investigate the effects of an oral formulation of cannabidiol. The purpose of this Phase III study is to see if this investigational drug is safe and how well it works in controlling seizures associated with DS. The trial will include children who meet the eligibility requirements and are taking one or more seizure medications. Participants will receive either the investigational medicine or placebo (on top of their current medications) during the study. Participants who complete the study may have the opportunity to join a follow-up open label extension study and will receive study medication. No placebo will be used in the extension study.

Participants may be eligible to receive a stipend for completing study visits and/or reimbursement of their study-associated travel costs, as determined by the site. For more information on this study and patient eligibility, please review the listing with your physician and have them contact GW Pharmaceuticals at