ABOUT THE FAiRE-CDD STUDY
Fenfluramine has been tested in patients with two other types of epilepsy, one called Dravet Syndrome and the other called Lennox-Gastaut Syndrome (LGS). Based on the positive safety and efficacy results from these studies, fenfluramine is now approved for Dravet syndrome in the US and several other countries, and it is currently under regulatory review for LGS approval. As a result of the previous clinical studies it is thought that Fenfluramine may work in two ways. First, it causes the release of serotonin, which is a brain chemical involved in regulation of emotions, sleep, anxiety, and other body functions. Second, it also affects the activity of sigma-1 receptors on nerve cells, which are thought to have a role in decreasing seizure activity.
There has been some limited previous clinical trial testing of how fenfluramine works to control seizures involving six patients between 2 and 26 years of age who had CDKL5 Deficiency Disorder (CDD) and were treated with fenfluramine for an average of about 5 months. The results of this trial were promising in that these patients experienced a decrease in the number of seizures they had compared to when they began treatment.
This study is open to male and female patients, between the ages of 1 and 35, who have been diagnosed with CDD using both a genetic test (which can determine if there is a mutation or mistake in the CDKL5 gene) and through a clinical diagnosis by a doctor. Some patients who have been diagnosed with CDD have also been diagnosed as having LGS in the past because both disorders have some things in common including a lot of seizure activity. These patients are also eligible for this study.