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 CLINICAL TRIALS FOR DRAVET SYNDROME
FAiRE Trial (Fenfluramine Assessment in Rare Epilepsy) 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 firstname.lastname@example.org
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.gov listing with your physician and have them contact GW Pharmaceuticals at email@example.com
PTC 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. Please feel free to contact the study coordinator, Latoya King, if you have any further questions.