FAiRE is a clinical study program for children and young adults with Dravet syndrome. On their website, they provide information and an opportunity to get involved and be a study participant if interested. Check out their website here.
What is Dravet syndrome?
Dravet syndrome is a very serious form of epilepsy that starts in infancy. This is a rare disease that occurs in only 1 out of every 20,000-40,000 births.
Dravet syndrome can cause seizures and behavioral and developmental problems. This condition can cause issues with balance and speech as well. Growth issues and problems sleeping can occur as well as infections and issues with the nervous system. Certain anti-seizure medications can reduce the number of seizures for a patient. However, this does not work for all patients and this is why studies like FAiRE are so important.
FAiRE supports clinical studies or trials to find treatment for Dravet Syndrome. Clinical trials or studies help researchers study new medications or improve medicines that are already available.
Through a clinical study or clinical trial, researchers can determine if an investigational drug works, if it’s any better than medicine already on the market, and if it has any side effects.
Studies have risks that cannot always be determined or predicted. Participants of a clinical study must agree to a schedule of clinic visits and telephone appointments so their health and improvement can be monitored while on treatment.
FAiRE stands for Fenfluramine Assessment in Rare Epilepsy. FAiRE focuses on conducting clinical studies to see if the investigational drug called ZX009 improves seizures in young adults and children with Dravet syndrome.
The purpose is to determine if ZX009 is able to reduce the frequency of seizures compared to other anti-seizure medicine and to see what side effects it might cause.
ZX009 is a solution of low dose fenfluramine by mouth.
In the past, ZX009 had issues with causing serious heart related side effects. Now, lower doses have been given to some patients (for as long as 26 years) with no signs of heart disease.
There are several options for participants to join a clinical study. Some of the studies last 22 weeks and some last for 26 weeks. One group of participants will receive the ZX009 medicine and the second group will randomly receive placebo pills. All participants will have their health checked regularly.
After the testing period, participants will have a follow up period where their health will be monitored to see what happens after the dosing plan.
