Participate in Research
Are you interested in having your family member with Dravet syndrome or a related ion channel epilepsy participate in research studies? These studies might range from an online survey or phone interview, to blood tests and other medical procedures.
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. Along with clinical trials, DSF supports and endorses four registries and highly encourages families to participate wherever they can.
Zogenix Trial of ZX008 (Fenfluramine HCL) in Children & Young Adults with Dravet syndrome – Now Enrolling!
This is a multicenter, double-blind, parallel-group, placebo-controlled, study 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. After an initial Screening and Baseline charting of seizure frequency, subjects who qualify for the study will be randomized (1:1:1) to receive either ZX008 (0.2 mg/kg/day, 0.8 mg/kg/day; maximum dose: 30 mg/day) or placebo. Randomization will be stratified by age group (< 6 years, ≥6 to 18 years). All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Subjects exiting the study will undergo a 2-week taper, unless they enroll in a follow-on study. Subjects will be followed for post-study safety monitoring. Parents/caregivers will use a diary daily to record the number/type of seizures, dosing, and use of rescue medication.
Additional details and study center information on sites in the U.S. and Canada may be found at clinicaltrials.gov Please make sure to contact the study coordinator at the site you are interested in, and not the physician, to see if your child is eligible. For information about details related to the ZX008 phase 3 program, please contact Zogenix Medical Affairs at email@example.com
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 clinicaltrials.govlisting with your physician and have them contact GW Pharmaceuticals at firstname.lastname@example.org
IICEPR (International Ion Channel Patient Registry) Clinical Registry
University of Michigan Department of Neurology & Miami Children’s Hospital Brain Institute
Principal Investigators: Jack M. Parent, MD & Ian Miller, MD
Funded by DSF & ICE Epilepsy Alliance
IICEPR is the only clinical registry for ion channel epilepsy patients where medical information and genetic test results are collected and stored in a standardized and secure way by medical professionals. This tool is available to all researchers for use in research & clinical studies.
IICEPR is the only clinical registry for ion channel epilepsy patients where medical information and genetic test results are collected and stored in a standardized and secure way by medical professionals. This tool is available to all researchers for use in basic science and clinical research studies.
To join the IICEPR, click here Participants will be contacted directly by IICEPR should they qualify to participate in a clinical study
DSF Dravet Family Network
DSF’s Dravet Family Network is a communication tool that will provide you with the most up-to-date information about research projects and clinical trials that are available for participation. Families will also be notified if there is a new family near them who wants to connect with the community or if there are fundraising or awareness events near them. Patients and their families enrolled in this network will receive periodic updates. Your information will not be shared without your permission; the network is simply a tool for communication within the Dravet community.
To join DSF Dravet Family Network, click here
Rare Epilepsy Network (REN) Registry
The Rare Epilepsy Network, or REN for short, is a collaboration between the Epilepsy Foundation, the IFCR, RTI international, Columbia University, Dravet Syndrome Foundation and many different organizations that represent patients with a rare syndrome or disorder that is associated with epilepsy or seizures. The REN will establish a registry of these patients which includes patient or caregiver-reported data in order to conduct patient-centered research. This research will be in the form of natural history studies and completion of surveys. The registry will also create the infrastructure for future research such as clinical trials. All of the research will be patient-centered, which means it will address research questions and topics that are important to the patients and caregivers with the ultimate goal of having patients and caregivers better able to participate in healthcare decisions. You may stop participating in the research at any time.
What Do We Mean by “Rare Epilepsy”?
In the world of neurology, “rare epilepsy” is a disorder or syndrome that is defined by a particular type of seizure. For example, Dravet syndrome is also known as Severe Myoclonic Epilepsy of Infancy. For the purposes of the Rare Epilepsy Network, we are including in the definition of rare epilepsy any disorder that is rare and that is also associated with seizures or epilepsy in the majority of patients. We are also using the term epilepsy to mean the same thing as having seizures or a history of seizures.
Who is Eligible for the Registry?
Patients who are eligible for the REN Registry must have a diagnosis by a physician of one of the following conditions:
Eligible patients must also have had at least one seizure in their lifetime that was not caused by a fever or the direct result of a head injury. We call these types of seizures “unprovoked”. DSF is hoping to recruit at least 250 people with Dravet syndrome into the REN.
What Do I Need to Do to Participate?
Enrollment is easy! There are 3 steps to enrolling in the REN Registry:
- You will need to read a consent form and acknowledge that you understand what it says and you would like to participate. If you have any questions, you may send us an email or call us before you agree to participate.
- You will need to enter in your contact information and some information about the affected person which allows us to create a global unique identifier and to be able to contact you in the future about the Registry.
- You will need to complete an enrollment survey which asks questions about the affected person with Dravet syndrome which asks about their diagnosis, seizures, treatment, development and medical history. There is also a section that asks about your quality of life and the impact that epilepsy has had on your family. The more information you provide in completing the questions, the more valuable your data will be for research. If you have a list of seizure drugs that were EVER taken, this may help you in completing the section about treatment.
The survey can be completed all at once or in sections.
How Much Time will it Take to Participate?
Reading and signing the consent and filling out your contact information will take about 5-10 minutes. The entire enrollment survey will take about 45 minutes to complete but does not have to be completed all at once. It is divided up into sections. A few times a year we will send you an email asking you to complete a follow-up survey.
How Can I Learn More and Enroll in the Registry?
You may learn more about the Registry and enroll by logging on to the study website
SUDEP Tissue Bank Registry
DSF supports Dr. Alica Goldman and her colleagues at The Department of Neurology at Baylor College of Medicine in their establishment of a seizure disorder tissue bank, the SUDEP Tissue Donation Program (STOP SUDEP). This project is funded by The National Institutes of Health (NIH)/The National Institute for Neurological Disorders and Strokes (NINDS). Investigators are collecting blood and/or tissue samples from individuals who have passed away as a result of a seizure disorder.
To register for tissue donation, contact Dr. Alica Goldman