CATE Trials – Investigating MRX2 & MRX2T for Refractory Epilepsies

The aim of this research is to trial two novel cannabinoid medicines; MRX2 (CBD) and MRX2T (CBD in combination with THC), to determine whether they are effective and safe in a broad range of seizure types and epilepsies in children and adults.

• To investigate whether MRX2 and MRX2T are effective in reducing seizures in a broad range of epilepsies and across a broad range of seizure types.
• To investigate whether MRX2 and MRX2T have any impact on cognition, quality of life, level of behaviour and anxiety, sleep quality and parental stress.
• To estimate the cost-effectiveness of MRX2 and MRX2T from the perspective of the National Health Service and Personal Social Services.

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Two Phase 3a, double-blind, randomised controlled trials will be conducted in parallel. 

1. CATE-E : A double blind, Phase 3a randomised controlled trial of MRX2 and MRX2T versus placebo in patients with refractory early-onset Epilepsy “CATE-E”.
2. CATE-G: A double blind, Phase 3a randomised controlled trial of MRX2 and MRX2T versus placebo in patients with refractory genetic generalised epilepsy of normal cognitive ability “CATE-G”. 

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About Epilepsy and its treatment

Condition

Epilepsy is one of the most common neurological disorders affecting 1-2% of the population³. NICE have estimated that the combined direct and indirect costs to the National Health Service (NHS) are £2 billion per annum and most of this is spent on patients with refractory epilepsy. Refractory epilepsy is defined by the International League Against Epilepsy as a failure to achieve sustained seizure freedom after adequate trials of two tolerated and appropriately chosen antiseizure medications (ASMs). Approximately one third of patients with epilepsy will meet this definition⁴. Refractory epilepsy impairs quality of life, has significant health economic costs, causes individual and family stress, and may contribute to early mortality. 

There is, therefore, always a need to develop new effective and safe therapies that may ameliorate the refractory epilepsies and consequently improve the quality of life of patients and their families, reduce economic burden to the NHS, and potentially improve life expectancy.

Current treatment

In the last 30 years there has been an increase in the number of (ASMs) available to clinicians with >25 ASMs now licensed. Despite the number of drugs available there has been little change in the proportion of intractable cases⁵. Understandably, therefore, physicians, patients, parents and families are anxious to find any a treatment medicine that can potentially bridge this gap. 

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Trial Team

Helen Cross

Professor Helen Cross is The Prince of Wales’s Chair of Childhood Epilepsy and Head of the Developmental Neuroscience Programme at UCL-Great Ormond Street Institute of Child Health, Honorary Consultant in Paediatric Neurology Great Ormond Street Hospital for Children NHS Foundation Trust, London and Young Epilepsy, Lingfield, UK.

Helen's research has been targeted at improving outcomes in early onset epilepsy, specifically in assessing the role of surgery and ketogenic diet. Helen has also held key leadership roles both nationally and internationally.

Helen is Treasurer of the International League Against Epilepsy 2017-2021 (Secretary General 2013-2017), Clinical Advisor to the National Children’s Epilepsy Surgery Service, and Clinical Advisor to the update of the NICE guidelines for Childhood Epilepsy 2018-2021. She developed, as Coordinator, the European Reference Network for Rare and Complex Epilepsies (EpiCARE) launched in 2017.

Finbar O’Callaghan

Professor Finbar O'Callaghan is Professor of Paediatric Neuroscience at the Institute of Child Health, University College London.

He has been a Consultant Paediatric Neurologist at Great Ormond Street Hospital since September 2013 and prior to that was a Consultant at Bristol Royal Hospital for Sick Children for 10 years.  Finbar was also the Chair of the British Paediatric Neurologists Association.

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