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
Transition From Pediatric to Adult Epilepsy Care
The goal of this observational study is to assess the preparation of the caregiver and the patient for the transition, by comparing the results of the Transition Readiness Assessment (TRAQ) questionnaire at the time of the last pediatric neuropsychiatric visit, which is then repeated at the first adult epilepsy neurology visit. The assessments will be conducted at the last evaluation at the pediatric epilepsy service and repeated at the first visit to the adult epilepsy service.
A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome
This is an open-label, single-center study to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of intrathecal RC001 in patients with Dravet syndrome aged 2 to 18 years. The study includes a dose-escalation part followed by a fixed dose treatment part, with participant progression based on investigator-assessed safety and efficacy.