Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT03572933) enrolled 101 people in total — 51 received a placebo (a dummy treatment with no active ingredient) and 50 received ganaxolone, a medicine being studied for a type of epilepsy involving major motor seizures (seizures that cause strong physical movements such as stiffening, jerking, or sudden falls). The trial compared how often these seizures occurred over a double-blind treatment period (meaning neither the participants nor the researchers knew who was receiving which treatment) relative to a six-week period of observation before treatment began. By the end, 47 placebo participants and 48 ganaxolone participants completed the study. The reported data shows that, when looking at the main (primary) outcome — the number of major motor seizures per 28 days — the placebo group started at a median of roughly 49 seizures per 28 days at baseline and this figure was around 56 during the treatment period. The ganaxolone group started at around 54 seizures per 28 days at baseline and this figure was around 45 during the treatment period. For the secondary outcomes, the percentage of days completely free of major motor seizures was reported as approximately 36% for the placebo group and 32% for the ganaxolone group during the treatment period. Caregivers and clinicians also rated changes in attention, behaviour, and overall impression of improvement on structured rating scales; the reported counts across the different rating categories are available in the full submission, but no single summary figure clearly favoured one group over the other in a straightforward way based on the numbers provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 36517284) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Epilepsy Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have a confirmed genetic mutation in the CDKL5 gene, started having seizures before age 1, and were not able to walk independently by age 2
- Seizures have not been well controlled even after trying 2 or more anti-seizure medications
- You have at least 16 seizures of the main seizure type within every 28-day period
- You are currently on a steady dose of between 0 and 4 anti-seizure medications (a vagus nerve stimulator, ketogenic diet, or modified Atkins diet do not count toward this number)
- There are additional requirements to join that the study team can explain to you
Who may not be able to join:
- You have previously taken the study drug, ganaxolone
- You have a specific seizure condition called West Syndrome with a particular abnormal brainwave pattern, or your seizures are mostly of the "infantile spasms" type
- You are currently taking certain steroids (such as ACTH or prednisone) or certain other medications that can affect how drugs are processed by the body (confirm with trial site)
- You are using marijuana (THC) or CBD products, unless you have a prescription for a specific FDA-approved CBD medication called Epidiolex®, during the main part of the study
- You have taken part in another clinical trial or taken another experimental drug within the last 30 days or a certain short time period before screening
- A specific hormone level in your blood (called Allo-S) was found to be too high at your first screening visit (confirm with trial site)
- There are additional reasons that might prevent joining, which the study team can discuss with you
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Joseph Hulihan, MD, Marinus Pharmaceuticals, Inc.
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Summary of 28-day Seizure Frequency for Major Motor Seizure Types
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.