A Multicenter, Open-Label, Long-Term, Safety and Tolerability Extension Study of PTI5803 as adjunctive therapy in patients ≥ 14 years of age with drug-resistant seizures associated to focal cortical dysplasia.
Focal cortical dysplasia is an area of the brain that did not develop normally before birth, which can trigger seizures that are hard to control with standard medicines. This clinical trial is an extension study testing an add-on medicine called PTI5803. It is designed for people who took part in an earlier study of this treatment. The main goal of the trial is to check if taking PTI5803 over a longer time is safe and easy to tolerate. Doctors will look for side effects and check general health, heart rhythms, and mood. Researchers will also use daily diaries to track how often seizures happen and see if patients feel their quality of life improves. Taking part gives researchers vital information about whether PTI5803 could be a helpful long-term option for people living with drug-resistant epilepsy.
At a glance
What is this study about?
Some people have a type of epilepsy linked to focal cortical dysplasia. This means a small area of the brain did not form properly before birth. For many people with this condition, standard epilepsy medicines do not stop their seizures. When treatments fail to control seizures, doctors call this 'drug-resistant' epilepsy, and finding new options is very important.
This study is testing an experimental medicine called PTI5803. It is taken alongside a person's usual anti-seizure medicines as an add-on treatment. Because this is an 'extension study', it is designed for patients who have already completed an earlier trial with this drug. Researchers want to learn what happens when people take PTI5803 over several months or years.
Safety is the main focus of the trial. Doctors will closely check how the body handles the medicine by carrying out regular health checks, blood tests, and heart tracings. They will also track changes in mood and emotional wellbeing. At the same time, researchers will see if the medicine reduces the number of seizures people have and helps them feel better in their day-to-day lives.
Key takeaways
- Tests an add-on medicine called PTI5803 for hard-to-treat epilepsy.
- Aimed at people whose seizures are caused by focal cortical dysplasia.
- An extension study for people who completed the previous PTI5803 trial.
- Open-label study, meaning all participants receive the active medicine.
- Monitors long-term safety, mood, seizure numbers, and quality of life for up to two years.
Who may be eligible?
This study is specifically for people who have drug-resistant focal epilepsy caused by focal cortical dysplasia. Because this is an extension study, participants must usually have taken part in the earlier trial (called study A_CL_002) of PTI5803.
The trial is open to both men and women. While the wider study includes young people aged 14 and over, some hospital sites may only enrol adults aged 18 and over. Your study doctor can confirm the exact age rules for your local clinic.
To take part, you will need to continue taking your regular epilepsy medicines as advised, attend scheduled hospital appointments, and be able to keep a daily diary of any seizures you experience.
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.
- You have focal epilepsy linked to focal cortical dysplasia.
- Standard epilepsy medicines have not fully controlled your seizures.
- You previously completed the earlier trial of PTI5803 (study A_CL_002).
- You meet the age criteria for your local clinic (either 14+ or 18+).
- You are willing to attend regular clinic check-ups and keep a daily seizure diary.
What does participation involve?
Taking part involves taking the study medicine, PTI5803, alongside your usual epilepsy treatments for up to two years. You will attend regular check-ups at the hospital clinic. During these visits, the study team will measure your blood pressure, weight, and height, and check your heart rhythm with an ECG. They will take routine blood and urine samples and ask you questions about your mood, emotional health, and everyday quality of life. You will also be asked to keep a daily diary to record the number and type of any seizures you have. These visits help the team watch your health closely and ensure the medicine remains safe for you.
Potential risks and benefits
Locations (1)
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Common questions
What is focal cortical dysplasia?
It is a condition where a small part of the brain did not develop normally before birth. It is a common cause of epilepsy that does not respond well to ordinary medicines.
Do I have to stop taking my normal epilepsy tablets?
No. PTI5803 is an 'add-on' treatment, meaning you take it together with your current prescribed anti-seizure medicines.
Will I definitely receive the real medicine?
Yes. This is an 'open-label' study, which means there is no dummy pill (placebo). Everyone who joins will receive PTI5803.
How long will I be in the study?
The study looks at long-term safety, and you may be followed by the medical team for up to 24 months (two years).
What happens if I change my mind about taking part?
You can leave the trial at any point. Your medical team will support you and discuss the best way to return to your standard care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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