UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome v1
Research type
Research Study
Full title
UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM) This is the UK arm of EU study “Multinational Européan Trial for Children with the Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome“.
IRAS ID
119677
Contact name
Michael Pike
Contact email
Sponsor organisation
Oxford University Hospitals NHS Foundation Trust
Eudract number
2011-000990-29
Research summary
Opsoclonus myoclonus syndrome (OMS) is a rare disorder of the nervous system (incidence 1/5 million/year) with onset usually in the second year of life. It presents as jerky movements of the eye (opsoclonus) and body (myoclonus), with ataxia, irritability and sleep disturbance, and is associated with subsequent learning, movement and behavioural problems. About 50% of children with OMS have an underlying neuroblastoma and it seems likely that it is an immune-mediated, sometimes paraneoplastic, condition. Steroids, often supplemented with other immunosuppressants, are the primary treatment but there is limited evidence for drug choice and dosage and little knowledge of the relationship between early symptomatic response and later cognitive outcome. This study will examine drug response of OMS children, with and without NB. 100 children (15 from the UK), recruited over 3 years across 8 European countries, will be treated with an escalating 3-step schedule. All will receive a pulse of dexamethasone (3 consecutive daily doses) at diagnosis and then at a further eleven 4-weekly intervals. Children who fail to show marked benefit after three pulses of dexamethasone will additionally receive cyclophosphamide at each of the next four or six dexamethasone pulses. Those who show inadequate improvement after 4 doses of cyclophosphamide will in its place receive two doses of rituximab at 2-weekly intervals. The primary outcome measure will be remission of OMS symptoms/signs as recorded by the clinician. For each treatment group the final statistic will be percentage of participants in disease remission. Secondary outcome measures will be improvement in OMS symptom score and cognitive and behavioural outcome. A European bio-bank will be established.
Lay summary of study results: This is a European study and data from the other 7 nation states involved are continuing to be collected in the central European database. The data includes Opsoclonus Myoclonus Syndrome (OMS)/Dancing Eye presenting features, descriptions of the neuroblastoma tumour if present, treatment pathways, neurological evaluation and follow-up including cognitive and behavioural assessment, as well as details of any adverse events or serious adverse events. This data will contribute to information gathered on OMS and neuroblastoma tumour studies in general, enabling the sharing of information across the research network to harmonise standard procedures and integrate research efforts.
A biobank has been established at Oxford University Hospitals Trust to store the samples collected from participants with guardian consent. Some of these samples have already been analysed and linked to clinical data, leading to the characterisation of a novel and potentially clinically relevant auto-antibody. Detection of this antibody has possible utility as a diagnostic test for OMS. ( Berridge G, Menassa DA, Moloney T, Waters PJ, Welding I, Thomsen S, Lang B. (2018). Glutamate receptor δ2 serum antibodies in pediatric opsoclonus myoclonus ataxia syndrome. Neurology, 91(8), pp. e714-e723. doi: 10.1212/wnl.0000000000006035). Further studies utilising the research biobank will be undertaken to clarify the pathological relevance of this novel autoantibody. The biobank is open for all researchers to apply to use the samples stored in the future.
REC name
London - Fulham Research Ethics Committee
REC reference
13/LO/0706
Date of REC Opinion
9 Jul 2013
REC opinion
Further Information Favourable Opinion