RCT in children with ESES syndrome
Research type
Research Study
Full title
Corticosteroids or clobazam for ESES syndrome: a European, multicenter, randomized, controlled clinical trial
IRAS ID
161657
Contact name
Bart van den Munckhof
Contact email
Sponsor organisation
University Medical Center Utrecht
Eudract number
2013-000531-27
ISRCTN Number
ISRCTN42686094
Clinicaltrials.gov Identifier
43510, Dutch Central Committee on Reasearch involving Human subjects (CCMO) Registration number (ABR)
Duration of Study in the UK
3 years, 2 months, 29 days
Research summary
Background: Electrical Status Epilepticus in Sleep (ESES) is a rare subclinical electroencephalographic (EEG) pattern of sleep-induced spikes and waves (SWs) in children. This typical EEG pattern is characterized by SWs during >85% of slow-wave sleep, called ESES. Epileptic encephalopathy with ESES is defined by - often dramatic - language, cognitive, and behavioral problems, further called ESES syndrome . Although the ESES itself usually resolves during puberty, cognitive deficits persist into adulthood in the majority of patients, especially when ESES has not been treated. The major goal of treatment in patients with ESES syndrome is to improve the associated cognitive deficits. Unfortunately, the effects of conventional anti-epileptic drugs are extremely disappointing, except for clobazam, with a reported response rate of around 30%. Corticocorticosteroids may be more effective than clobazam, but these are not without risk and treatment strategies have never been compared in a randomized controlled clinical trial.
Objectives: The primary objective is to compare the effects on cognition of treatment with corticosteroids and clobazam in children with ESES. Secondary objectives include to compare the effects of these treatments on the index of sleep induced SWs (SWI) on EEG, the incidence of seizures, safety, to assess which patients benefit most from therapy, including study of activation of the immune system as potential biomarkers .
Methods: This is a European, multicenter, randomized, controlled, clinical trial with blinded outcome assessment in 130 patients with ESES syndrome, to compare the effects of treatment with treatment with corticosteroids, allowing two different administration regimens, for a period of six months and daily clobazam. Patients may be included if they are between 2 and 12 years of age and have been diagnosed with typical or atypical ESES syndrome in the previous 6 months.REC name
London - Brent Research Ethics Committee
REC reference
14/LO/1857
Date of REC Opinion
10 Dec 2014
REC opinion
Further Information Favourable Opinion