Cavernomas: A Randomised Effectiveness (CARE) Study
(This Research study is now closed to new participants)
Purpose of the CARE Study
This research study wants to find out what approach is best for treatment of people with a brain cavernoma that has caused symptoms like stroke or epileptic seizure.
In this short video, the CARE Study’s chief investigator – Professor Rustam Al-Shahi Salman – explains what this ‘pilot study’ is all about…
A cavernoma is a cluster of blood vessels that form blood-filled ‘caverns’ that look like a raspberry. Brain cavernomas can cause strokes, epileptic seizures and other symptoms related to these two problems.
- The problems caused by a stroke from a cavernoma depend on its location in your brain. These symptoms are usually mild, but sometimes they cause disability, other health or social problems or, very rarely, death.
- An epileptic seizure caused by a brain cavernoma may be a one-off; if it happens more than once, this is known as epilepsy. Epileptic seizures can cause disability (e.g. loss of your driving licence), other health or social problems or, extremely rarely, death.
Standard treatment without surgery for most people with brain cavernoma involves treating their symptoms, preventing seizures with drugs, or helping recovery with rehabilitation. About one fifth of people in the UK with brain cavernoma have treatment including surgery, involving standard treatment and either neurosurgery or stereotactic radiosurgery (usually using Gamma Knife).
But we’re not sure which treatment approach is best for many people with a symptomatic brain cavernoma. For some people, doctors are certain about recommending standard treatment of symptoms without surgery. For some others, doctors are certain about recommending treatment of the cavernoma including surgery as well. But for many people with cavernomas, doctors are uncertain about what treatment to recommend.
The risks and benefits of treatment without surgery seem similar to treatment including surgery. There is no reliable evidence about which is better. This can leave doctors and patients uncertain about whether to use surgery. Answering this uncertainty is the top priority for research into cavernoma, and the purpose of this study.
Why is CARE a Pilot Study?
Finding an answer to the question “How effective is treatment with neurosurgery or stereotactic radiosurgery versus treatment without surgery in people with symptomatic brain cavernoma?” requires a special type of research study known as a Randomised Controlled Trial.
It is estimated that about 1,000 people will be needed to answer this question about symptomatic brain cavernoma. This is more than are likely to be found in the United Kingdom and Ireland alone, even by a study lasting several years. The latest figures suggest that there are about 180 new symptomatic brain cavernoma diagnoses each year in the UK, and an unknown fraction of these would be recruited to this study. This means that a main study will need to be international, recruiting in many countries across the world.
Therefore the CARE Study is a first step to determine whether it will be feasible to do the main definitive study. The CARE Study is a piece of research done before the main study in order to answer the question, “Can this study be done?” It will estimate important parameters that are needed to design the main study, including what fraction of patients who are diagnosed each year will wish to take part. The CARE Study includes a ‘pilot study’, which is a version of the main study that is run in miniature to test whether the components of the main study can all work together. It is focused on the processes of the main study, for example to ensure recruitment, randomisation, treatment, and follow-up assessments all run smoothly. Therefore, the CARE Study resembles what the main study is likely to be like.
What does the CARE Study involve?
- The Randomisation Study is comparing treatment without surgery to treatment including surgery for people with symptoms from brain cavernoma.
This is the pilot study. It involves a special type of research called a, “randomised controlled trial”, which is the most reliable test of treatments. More information about this type of research is available. A patient’s treatment in this study will take place in one of up to thirty-nine Study Sites in the UK and Ireland. These are located within a Neuroscience Centre at a hospital. Each Study Site has a clinical Research Team, members of whom will be involved in your care alongside your own doctors, if you take part in this study.
- The Information Study, is integrated within CARE and intends to improve how we give information about research to patients and learn what patients (and the parents of affected children) think about taking part. This simply involves audio-recording the conversations patients or families have with doctors about the CARE study and inviting patients/family members to take part in an interview asking for their thoughts and experiences of the study. This helps the CARE study team learn how to provide information about the CARE study, in a way that meets patient needs.
These two parts of the CARE Study will tell us if enough eligible patients take part to indicate that a larger definitive study could be done to resolve the uncertainty about treatment of symptomatic brain cavernoma.
These are described more fully in the Patient Information Leaflets. The CARE Study is now closed to enrollment of new patients. If you are an enrolled patient and have a question please get in touch with us through the Contact Us page.
The support Cavernoma Alliance UK can provide
CAUK is the patient group co-applicant in the study and has been involved in its development by providing the patient voice from the start. We have co-developed the information leaflets for patients.
We also coordinate a Patient Advisory Group to oversee the conduct and progress of the CARE Study.