Study into treatment of children with difficult to treat epilepsy due to Tuberous sclerosis complex with Rapamycin.
Completed
- Conditions
- TSCTuberous sclerosis complexTubereuze sclerose complexRapamycinRapamycineEpilepsyEpilepsiemTORmTORC1
- Registration Number
- NL-OMON28870
- Lead Sponsor
- Erasmus University Medical Center
- Brief Summary
/A
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 23
Inclusion Criteria
1. Children with definite TSC;
2. Older than 3 months, up to 12 yrs old;
Exclusion Criteria
1. Renal dysfunction;
2. Surgery during 6wk before inclusion;
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary outcome is change in frequency of epileptic seizures. This will be assessed by comparing the seizure frequency at baseline to the seizure frequency after 6 months of rapamycin added to the standard care. Seizure frequency is recorded by the parents, using an epilepsy diary.
- Secondary Outcome Measures
Name Time Method 1. Proportion of responders (seizure reduction of 50% or more);<br /><br>2. Seizure severity;<br /><br>3. EEG changes;<br /><br>4. Psychomotor development;<br /><br>5. Side effects.