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
Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (EPIC (EPIlepsy Cell Therapy))
This clinical trial is designed to test whether a single image-guided intracerebral administration of inhibitory nerve cells, called interneurons (NRTX-1001), into subjects with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), with or without mesial temporal sclerosis (MTS), is safe (frequency of adverse events) and effective (seizure frequency). NRTX-1001 comprises human interneurons that secrete a neurotransmitter, gamma-aminobutyric acid (GABA).
Collaborative Care for Anxiety and Depression in Epilepsy
This is a randomized effectiveness/implementation trial comparing a 24-week neurology-based collaborative care intervention to usual neurology care among 60 adults with epilepsy.