Brain cavernoma Nervous System Diseases
Conditions
Interventions
Current interventions as of 28/10/2022:
This prospective randomised open blinded end-point (PROBE) randomised controlled trial (RCT) aims to estimate the feasibility of performing a definitive main ph
Sponsors
University of Edinburgh
NHS Lothian
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. People of any age 2. At least one brain cavernoma diagnosed by brain MRI that included a gradient echo or susceptibility-weighted sequence, according to standard diagnostic criteria 3. Clinical history attributable to a brain cavernoma of: 3.1. Symptomatic stroke due to intracranial haemorrhage, or 3.2. Symptomatic stroke due to a persistent or progressive non-haemorrhagic, or not otherwise specified, focal neurological deficit, or 3.3. Epileptic seizure(s) meeting the definition of definite or probable cavernoma-related epilepsy 4. Patient and doctor are uncertain about medical management or medical and surgical management of the symptomatic brain cavernoma, following consultation with a neurosurgeon 5. Patient has mental capacity to consent for themselves (adult participants or paediatric participants with capacity) or parent/legal guardian provides consent (paediatric participants)
Exclusion criteria
Exclusion criteria: Current exclusion criteria as of 28/10/2022: 1. Surgical management of a solitary symptomatic brain cavernoma with MRI evidence of cavernoma removal/obliteration 2. Spinal cavernoma alone, without symptomatic brain cavernoma 3. Asymptomatic brain cavernoma. Patients with radiographic cavernoma enlargement (with or without intralesional haemorrhage) but without new symptoms are still regarded as asymptomatic 4. Previously randomised in the CARE pilot trial Previous exclusion criteria: 1. Surgical management of a solitary symptomatic brain cavernoma with MRI evidence of cavernoma removal/obliteration 2. Spinal cavernoma 3. Asymptomatic brain cavernoma. Patients with radiographic cavernoma enlargement (with or without intralesional haemorrhage) but without new symptoms are still regarded as asymptomatic 4. Previously randomised in the CARE pilot trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Feasibility measured using the following questions answered from the assessments performed and data collected at the baseline, 6-month local in-person follow-up and 6-monthly central follow-up: 1. What proportion of the collaborating centres take part and recruit participants to the CARE pilot trial? 2. Can the investigators implement trial procedures correctly? 3. What proportion of screened patients is eligible? 4. What proportions of eligible patients are approached and randomised (and why are eligible patients not approached or not randomised)? 5. What is the distribution of participants between neurosurgery and stereotactic radiosurgery? 6. Do participants adhere to the allocated intervention and follow-up? 7. How complete are baseline, imaging and outcome data? 8. What are the outcome event rates? 9. How do the baseline characteristics, outcome event rates and differences between treatment groups compare to observational data about outcomes during medical management or after medical and surgical management? 10. What estimates of effect size/variability should be used in the design of the CARE definitive main phase trial? 11. What is the sample size required for a definitive trial to address the overall question over a 10-year follow-up? 12. Can the CARE pilot trial data describe care pathways, linked to health states and outcomes, to develop a robust economic model to evaluate cost-effectiveness in a CARE definitive main phase trial? 13. Which international research partners in other countries could contribute to the CARE definitive main phase trial? Primary clinical outcome: Intracranial haemorrhage or new persistent/progressive focal neurological deficit due to brain cavernoma or surgical management (neurosurgery or stereotactic radiosurgery), whether fatal (leading to death within 30 days of the outcome event) or non-fatal measured using patient records at 6-monthly follow-up until the end of the trial | — |
Secondary
| Measure | Time frame |
|---|---|
| Measured at 6-monthly follow-up until the end of the trial: 1. Death not due to a primary clinical outcome measured using patient records 2. Seizure severity and frequency measured using the Liverpool Seizure Severity Scale plus epileptic seizure frequency (number of seizures in the preceding four weeks, and attainment of one-year seizure freedom) 3. Degree of disability or dependence in the daily activities measured using the Modified Rankin Scale (mRS) score 4. Impairment caused by stroke measured using the National Institute of Health Stroke Scale Score (adult or paediatric) 5. Quality of life measured using the EQ-5D-5L in adults and EQ-5D-Y in children 6. Functional status measured using the Karnofsky Performance Status (KPS) scale in adults and Lanksy Play-Performance Scale (LPPS) in children 7. Health service use and healthcare and socioeconomic costs measured from patient records | — |
Countries
England, Ireland, Northern Ireland, Scotland, United Kingdom, Wales
Outcome results
None listed