A double-blind, randomized, placebo-controlled study to evaluate the efficacy, safety, and tolerability of intravenous ganaxolone added to standard of care in refractory status epilepticus
This important study is looking into a new medicine, ganaxolone, for people experiencing a very serious and persistent type of seizure called refractory status epilepticus (RSE). RSE happens when seizures don't stop even after trying initial treatments. The main goal is to see if ganaxolone can stop these seizures within 30 minutes and prevent them from coming back for a certain period, without needing more treatment. Researchers will also be carefully checking for any side effects. This is a "Phase III" study, which means it’s one of the final steps before a medicine might be considered for wider use. Participants will be given either ganaxolone or a dummy medicine (a placebo) alongside their usual care, and neither they nor their doctors will know which one they are receiving.
At a glance
What is this study about?
This study is about helping people who have a very serious type of seizure called refractory status epilepticus (RSE). RSE means that someone is having continuous seizures or seizures that keep happening one after another, and these seizures haven't stopped even after doctors have given them the usual first treatments.
The main aim of this study is to test a new medicine called ganaxolone. Doctors want to find out if adding ganaxolone to the treatments people are already receiving can stop these difficult-to-control seizures quickly and keep them from coming back. They also want to make sure the medicine is safe and doesn't cause too many unwanted side effects. Finding effective treatments for RSE is very important because it truly is a medical emergency.
To make sure the results are fair, some people in the study will receive ganaxolone, while others will receive a dummy medicine called a placebo. Neither the patients nor their doctors will know who is getting which, until the study is over. This is called a "double-blind" study and it helps researchers get the most accurate information about how well ganaxolone works compared to standard care alone. This is a "Phase III" study, which is usually the last big step before a new medicine can be considered for approval.
Key takeaways
- This study is for adults with severe, ongoing seizures (refractory status epilepticus).
- It's testing a new medicine called ganaxolone, given alongside usual care.
- The main goal is to see if ganaxolone can quickly stop seizures and keep them from returning.
- Half of participants will get ganaxolone, half will get a dummy medicine (placebo).
- Safety is a top priority, and you'll be closely monitored for any side effects.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is for adults, aged 18 and over, who are experiencing refractory status epilepticus (RSE). This means their seizures have continued despite receiving initial treatments from the doctors.
There are other specific medical conditions and treatments that might mean someone can't join the study, but these details are usually discussed directly with the study doctor.
Generally, if you are an adult and are currently experiencing seizures that haven't responded to standard treatments, you might be considered for this study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Are you currently experiencing seizures that haven't stopped with usual initial treatments?
- Are you able to receive medicines through a drip into your arm?
- Have your doctors advised that you have refractory status epilepticus (RSE)?
What does participation involve?
If you are eligible and agree to take part, you will be given either the study medicine, ganaxolone, or a dummy medicine (placebo) through a drip into your arm, in addition to your usual care. Neither you nor your doctors will know which one you are receiving.
Throughout the study, the medical team will carefully monitor your seizures, your general health, and look for any side effects. They'll regularly check your level of consciousness and how well you are responding. You will have assessments at different times, including 24, 36, and 72 hours after starting the medicine, and again when you leave the hospital or at your final check-up.
The total time you spend actively monitored in the hospital for the study will depend on how you respond to treatment, but the main observations for stopping seizures are made within the first few days. The total duration of your participation in the study from start to final follow-up will be discussed by your doctor.
Potential risks and benefits
Locations (14)
- —UnverifiedAustria
- —UnverifiedDenmark
- —UnverifiedFrance
- —UnverifiedLithuania
- —UnverifiedGermany
- —UnverifiedFinland
- —UnverifiedHungary
- —UnverifiedItaly
- —UnverifiedCroatia
- —UnverifiedSpain
- —UnverifiedBelgium
- —UnverifiedCzechia
Common questions
What is 'refractory status epilepticus'?
It's a serious medical emergency where seizures continue for a long time or keep happening without stopping, even after initial treatments.
What is ganaxolone?
It's a new medicine being tested to see if it can help stop these difficult-to-treat seizures.
What does 'double-blind' mean?
It means neither you nor your doctor will know if you're getting the study medicine or a dummy medicine, to keep the study fair.
Will I still receive my usual epilepsy treatment?
Yes, ganaxolone or the dummy medicine will be given in addition to the standard care you are already receiving.
How long will the study last for me?
The main part of monitoring is in the first few days, and your overall participation will be discussed by your doctor, depending on how you respond.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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