Epilepsy in tuberous sclerosis complex (TSC) MedDRA version: 17.0 Level: LLT Classification code 10032061 Term: Other forms of epilepsy System Organ Class: 100000004852
Conditions
Interventions
Trade Name: Vigabatrin (Sabril)
Product Name: vigabatrin
Pharmaceutical Form: Granules in sachet
Sponsors
The Children's Memorial Health Institute
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female infants with a definite diagnosis of TSC (Roach criteria; Roach 1998 or DNA confirmed), - Age up to 4 months at the moment of enrolment, - No clinical seizures seen by caregivers or on baseline videoEEG recording, - Written informed consent of caregivers. It is possible to give consent for the observational part of the study only. In this case, the child will not enter the randomized part of the study. Are the trial subjects under 18? yes Number of subjects for this age range: 100 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: - Any type of seizures observed till baseline visit, - Antiepileptic treatment at or prior to study entry, - Contraindications to MRI, - Any severe and/or uncontrolled medical condition that is considered by the investigator as possibly affecting the EPISTOP analyses or procedures.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC. ;Secondary Objective: Secondary objective of the clinical part of EPISTOP is to compare the effects of standard antiepileptic treatment in patients diagnosed as having epilepsy after clinical seizures vs after electroencephalographic epileptiform discharges, in a randomized trial in TSC patients. ;Primary end point(s): Primary endpoint of the study is the collection of a set of molecular and clinical biomarkers in full analysis set of patients, including all TSC infants participating in the study and the control subjects.;Timepoint(s) of evaluation of this end point: vEEG will be performed every 4 weeks (± 1 week) in the children under the age of 6 months, then every 6 weeks (± 1 week) in children under 12 months, and every 8 weeks (± 1 week) thereafter. MRI will be performed at baseline (unless patient had an MRI performed within 1 month before baseline visit and an additional scan cannot be performed without general anaesthesia) and then as clinically indicated, but not later than at the age of 2 years. Neuropsychological assessment using a specially designed targeted battery of test will be performed every 6 months. Blood samples will be collected at study entry, at the onset of epileptiform discharges on vEEG or at the age of 6 months, whichever is applicable, at the onset of clinical seizures and at the end of follow-up (age 2 years) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Key secondary endpoint is better efficacy of antiepileptic treatment in patients diagnosed as epileptic after electroencephalographic epileptiform discharges in comparison to patients diagnosed as having epilepsy after the onset of clinical seizures.;Timepoint(s) of evaluation of this end point: The parameters of efficacy assessment include: the distribution of seizure free patients, proportion of patients with drug resistant seizures, proportion of patients with normalized EEG, the neuropsychological outcome recognized as the results in a battery of tests performed at the age of 24 months. | — |
Countries
Belgium, Netherlands
Contacts
Public ContactEPISTOP Project Office
The Children's Memorial Health Institute
Outcome results
None listed