What the trial was testing
The trial enrolled 87 patients with epilepsy. The study was sponsored by Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
54% of children taking fenfluramine had their seizures cut in half or more, compared to only 5% taking a sugar pill.
JAMA neurology · 2020 · NCT02926898
These findings — that children taking fenfluramine had 54% fewer seizures per month than those on placebo — were published in the JAMA neurology and represent the headline result of the study.
Researchers tracked outcomes across 87 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with epilepsy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Fenfluramine (brand name Fintepla) is now FDA-approved for Dravet syndrome in patients 2 years and older. If your child has Dravet syndrome and seizures aren't well controlled with current medications, ask your neurologist whether fenfluramine might help.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open epilepsy trials
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
Home Ultra-long Term EEG Monitoring for Rare Epilepsies and Developmental and Epileptic Encephalopathies
With this study the Investigator expects to develop a precise patient-centered model of care by means of home ultra-long-term EEG monitoring with a minimally invasive wearable EEG device (sqEEG). The following aims will be pursued: 1. to assess the sensitivity, reliability, and safety of sqEEG to record seizures over prolonged periods; 2. to verify sensitivity and reliability of automated seizure detection algorithms and to assess circadian and ultradian seizure/interictal epileptic discharges; distribution for the development of personalized seizure action plan; 3. to evaluate whether data collected with sqEEG can improve the clinical management of the patients and treatment outcomes. The investigator expects to use this wearable at-home EEG device to obtain an objective quantification of electrographic and electro-clinical seizures over a twelve-week period up to twenty-four weeks at home. The precise quantification of seizures is essential for a tailored treatment approach and to effectively monitor the response to treatment adjustments. The potential innovation of this approach relies on the possibility of managing the patient at home, reducing the side effects related to hospitalization and objectively quantifying the disease burden in the real-life setting with the aim of improve globally the patients' quality of life.