Study type

Study topic

Human medicinal product
Disease /health condition

Study type

Non-interventional study

Scope of the study

Assessment of risk minimisation measure implementation or effectiveness
Effectiveness study (incl. comparative)
Safety study (incl. comparative)

Data collection methods

Primary data collection
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Study drug International non-proprietary name (INN) or common name

RUFINAMIDE

Anatomical Therapeutic Chemical (ATC) code

(N03A) ANTIEPILEPTICS
ANTIEPILEPTICS

Medical condition to be studied

Lennox-Gastaut syndrome
Population studied

Short description of the study population

Patients (aged ≥4 years) with Lennox-Gastaut Syndrome (LGS) who required a modification in anti-epileptic therapy (either the addition of another AED or the change of one drug to another); including patients who were already receiving rufinamide.

Age groups

Children (2 to < 12 years)
Adolescents (12 to < 18 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Special population of interest

Other

Special population of interest, other

Lennox-Gastaut Syndrome Patients

Estimated number of subjects

225
Study design details

Main study objective

Evaluation of safety during the use of rufinamide and other anti-epileptic drugs

Outcomes

Safety during the use of rufinamide and other anti-epileptic drugs: Evaluation of the incidence of seizures of medical significance (including status epilepticus, new / worsening of seizure types and withdrawal seizures) during exposure to anti-epileptic drugs, including rufinamide, in patients with LGS, Long term use of rufinamide, and other anti-epileptic drugs: Evaluation, within the constraints of this population, of the impact on maturation and development that anti-epileptic drugs, including rufinamide, has on the LGS population, seizure control in LGS patients, including those taking rufinamide and other anti-epileptic drugs, assessment of healthcare resource utilisation.

Data analysis plan

Seizure profile assessments (including seizures of medical significance, withdrawal seizures, and a generic seizure frequency scale) will be collected at each visit. Seizure data will be summarised using descriptive statistics and graphical displays of longitudinal data. If applicable, longitudinal models or regression models will be used to evaluate changes in seizure variables over time and by subgroups. Safety will be evaluated through the collection of AEs, and detailed information through structured questions on AEs of special interest (including suicidal-related events), and reasons for discontinuation of anti-epileptic drugs. Contrasting of AEs by anti-epileptic drug and combination will be on an ad hoc basis. Incidence rates of AEs will be displayed and Kaplan-Meier plots for key AEs will be presented. Developmental assessment (height and weight) and healthcare resource utilisation will also be performed.