FAiRE (Fenfluramine Assessment in Rare Epilepsy) is a program of clinical studies that are evaluating whether an investigational drug called ZX008, when taken in addition to current anti-epileptic treatments, can improve seizure control in children and young adults with rare epilepsies. Each study tracks seizure frequency, measures exposure to the drug, and assesses non-seizure related aspects of the condition, such as quality of life. Additionally, all participants will have their health checked regularly, both in person and over the telephone.
ZX008 is a low-dose formulation of fenfluramine taken by mouth twice a day, with or without food. Fenfluramine was marketed in the past as an appetite suppressant for obese adults and was used at higher doses than this study. It was withdrawn from the market in 1997 because some people developed serious heart-related side effects.
However, the doses used in the FAiRE program are 2 to 4 times lower than the previously marketed doses. To date more than 300 individuals with epilepsy have been treated with ZX008, some for over 25 years, and none have experienced any signs or symptoms of heart valve disease or pulmonary hypertension. Importantly, all study participants will be screened to ensure no pre-existing heart conditions are present, and the study includes an extensive heart-monitoring plan to help identify any early signs of heart-related side effects.
In each FAiRE study, participants will be given a dosing plan to follow for the duration of study participation. These dosing plans will differ between studies, but all participants will continue to take their existing anti-seizure medication(s) normally.