Encephalopathy with electrical status epilepticus in sleep, also called ESES syndrome MedDRA version: 20.0 Level: LLT Classification code 10032061 Term: Other forms of epilepsy System Organ Class: 100000004852
Conditions
Interventions
Trade Name: Frisium
Pharmaceutical Form: Tablet
INN or Proposed INN: CLOBAZAM
CAS Number: 22316-47-8
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceut
Sponsors
University Medical Center Utrecht
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age 2 up to 12 years (not including children of 12 years old); • A diagnosis within six months prior to study inclusion (preferably as close to inclusion as possible) of either: o Bilateral sleep-induced epileptiform activity with SWI in > 85% of nonREM sleep and developmental delay, arrest, or regression (“typical ESES syndrome”); o Arrest or regression of development and bilateral sleep-induced epileptiform activity with a SWI in > 50%, or unilateral sleep induced epileptiform activity with SWI in > 85% of nonREM sleep (“atypical ESES syndrome”); o Regression of development and unilateral epileptiform activity with a SWI of > 50% of nonREM sleep (“atypical ESES syndrome”); • No previous treatment with either corticosteroids or clobazam • No current treatment nor in the previous three months with carbamazepine, oxcarbazepine, vigabatrin, tiagabine, gabapentin and pregabalin; These drugs are known to possibly worsen the outcome in children with ESES syndrome and may therefore influence treatment result. Furthermore, these drugs can increase the SWI during sleep and may cause an electrographic pattern fulfilling the criteria for ESES. Inclusion of such cases with possible “treatment induced ESES” is not desirable. • Written informed consent by parents / legal representatives Are the trial subjects under 18? yes Number of subjects for this age range: 130 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Patients with a SWI during wakefulness of > 50% • Acute or chronic infectious disease (e.g. TB, HIV) • Immunodeficiency • Severe osteopenia/osteoporosis • Diabetes • Cushing syndrome • Severe respiratory insufficiency • Severe liver failure • Severe ulcera • Any other condition that, in the investigator’s judgement, contra-indicates the use of corticosteroids or clobazam
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the effects on cognition of treatment with either corticosteroids or clobazam in children with ESES syndrome;Secondary Objective: - To compare the effects of treatment with corticosteroids or clobazam on sleep induced spike-wave index in children with ESES syndrome. - To compare the effects of treatment with corticosteroids or clobazam on the frequency of seizures in children with ESES syndrome. - To compare the side-effects and tolerability of treatment with corticosteroids or clobazam in children with ESES syndrome. - To compare the effects of treatment with corticosteroids or clobazam in children with ESES syndrome as measured with a VAS score. - To assess demographic and disease-related predictive factors, including immunological factors, of succes of treatment with corticosteroids or clobazam in children with ESES syndrome. - To identify a biomarker for disease activity and treatment response.;Primary end point(s): - Intelligence quotient, or developmental quotient - Cognitive sumscore Improvement is defined as significant when improved by at least 75% of the standard deviation.;Timepoint(s) of evaluation of this end point: after six months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Individual absolute test results, and IQ scores; - Spike wave index during non-REM sleep. Improvement is defined as a rdecrease to less than 25%; - Seizure frequency. Improvement is defined as a reduction of 50% or more as compared with baseline; - Global improvement of functioning assessed with a VAS score (-5 to 5) - Safety and tolerability, as assessed by the occurrence of serious adverse events; - Differences in pro-inflammatory cytokine levels in patients with ESES who respond to either treatment strategies compared to nonresponders.;Timepoint(s) of evaluation of this end point: after six and 18 months | — |
Countries
Belgium, Denmark, Finland, France, Germany, Italy, Netherlands, Spain, United Kingdom
Contacts
Public ContactCoordinating investigator
University Medical Center Utrecht
Outcome results
None listed