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
Vigabatrin With High Dose Prednisolone Combination Therapy vs Vigabatrin Alone for Infantile Spasm
Infantile spasms (IS) are seizures associated with a severe infantile epileptic encephalopathy. Both cessation of spasms and electrographic response are necessary for the best neurodevelopmental outcomes. Adrenocorticotrophic hormone (ACTH), or prednisolone, or vigabatrin are considered the first-line treatment individually. However, ACTH expense and availability are the barriers in developing countries including Thailand. Vigabatrin, therefore, is the first recommended by Epilepsy Society of Thailand due to ACTH unavailability. Recently, combined steroid treatments (either ACTH or high dose prednisolone) with vigabatrin are superior in cessation of spasms compared to steroid treatment alone. Thus, this study is aimed to compare the efficacy of vigabatrin with high dose prednisolone combination therapy and vigabatrin alone.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research. Individuals affected by rare diseases, undiagnosed conditions, and disorders of unknown prevalence may voluntarily contribute health information to support natural history studies, participant recruitment, and other research efforts. Through partnerships with patient advocacy organizations and researchers worldwide, CoRDS helps connect rare disease communities with research opportunities while advancing knowledge across more than 7,000 rare diseases. Participation is free of charge.