What the trial was testing
The MARIGOLD enrolled 101 patients with epilepsy. The study was sponsored by Marinus Pharmaceuticals 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
Children taking ganaxolone had a 31% reduction in major seizures versus 7% with placebo.
The Lancet. Neurology · 2022 · NCT03572933
These findings — that kids taking ganaxolone had nearly one-third fewer major seizures than before treatment — were published in the The Lancet. Neurology and represent the headline result of the study.
Researchers tracked outcomes across 101 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
Ganaxolone received FDA approval for CDKL5 deficiency disorder in March 2022, shortly before this study was published. It's now available by prescription as Ztalmy. If your child has CDKL5-related seizures that aren't well controlled, ask your neurologist whether ganaxolone 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
RESting-state Functional MRI for Prediction of Post-surgical Prognosis In Pediatric Drug-Resistant Epilepsy
Resting-state functional MRI (r-fMRI) has emerged in recent years to analyze resting networks. It allows, without active participation of the patients, to identify and analyze the different functional brain networks. The analysis of r-fMRI can be done thanks to the graph theory, which is based on the based on the calculation of quantifiable parameters applied to the functional network studied, making it possible to assess its effectiveness. To the knowledge of the investigators, no study has used graph theory applied to r-fMRI data in order to obtain parameters useful for the useful parameters for the individual prognosis of children who have to be operation for drug-resistant lesional epilepsy.
Creation of a Database of Healthy Subjects With 18FDG PET Brain Imaging as Part of the MOBILE Project (Multimodal Whole-Brain Imaging in Epilepsy)
The MOBILE project is part of the dynamic European collaboration of the Human Brain Project (HBP). The overall aim of the project is to characterize brain structure and function in healthy subjects and patients with epilepsy, using a quantitative multimodal approach involving both neuroimaging (MRI, PET) and electrophysiology (EEG/MEG). The project is funded by the European HBP consortium, and the data acquired will ultimately be made available to the scientific community formed by this international collaboration. Several aspects of the project have already been initiated on the basis of extensions to previous authorizations, or as part of care activities. As part of this overall project, the present MOBILE-PET application concerns exclusively the performance of 18F-FDG PET (Positron Emission Tomography) imaging in the 30 healthy adult subjects in the protocol (aged 18 to 65, with inclusion parity for gender). This cerebral examination, performed at rest on a 45-minute 3D volume acquisition, enables quantitative measurement of the metabolic consumption of glucose underlying global synaptic activity, and to determine the associated connectivity. Around 1,500 examinations of this type are carried out each year in our department as part of care for patients with brain pathology, and over 10,000 for patients with cancer. This examination requires intravenous injection of a weakly radioactive tracer corresponding to a radiopharmaceutical which has been approved for marketing for over 20 years. We also carried out and finalized a similar project in 2007 on 60 healthy subjects, using a previous-generation PET camera (NCT00484523). The Nuclear Medicine Department holds clinical research authorizations for imaging in patients and healthy subjects (including early phase and first-in-man, although the present project does not fall into this research categarogy).