Placebo controlled study to assess the efficacy and safety of rapamycin in drug resistant epilepsy associated with tuberous sclerosis complex
- Conditions
- tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosisTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2021-004548-64-PL
- Lead Sponsor
- The Children's Memorial Health Institute
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 200
-male or female aged from 3 months up to 50 years at the day of randomization
-patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study
-patients/parents/caregivers are willing to and able to comply with all study requirements
-definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)
-drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks
Are the trial subjects under 18? yes
Number of subjects for this age range: 160
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
-history of treatment with mTOR inhibitor in the three months prior to screening,
-history of pseudo-epileptic seizures,
-history of progressive CNS disease other than TSC
-recent surgery within 2 weeks prior to the screening
-severe infection within 2 weeks prior to the screening
-use of the cannabis derivatives
-contraindications for MRI or general anesthesia
-occurrence of the serious comorbidities which, in the opinion of the investigator, may either put a patient at significant risk associated with the
participation in the study or may influence the results of the study the investigator
-pregnancy
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method