Tuberous Sclerosis Complex Epilepsy Tumors associated with Tuberous Sclerosis Complex
Conditions
Interventions
Trade Name: SABRIL 500 mg, granules for oral solution
Pharmaceutical Form: Granules for oral solution in sachet
Pharmaceutical form of the placebo: Oral powder
Route of administration of the placebo:
Sponsors
The Children's Memorial Health Institute
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: For the inclusion in the study, patients must fulfil all the following inclusion criteria: - Male or female aged from 4 up to 16 weeks (41-56 weeks of gestational age) at the day of randomization - Parents/caregivers are willing to and able to give informed consent form for the participation in the study - Parents/caregivers are willing to and able to comply with all study requirements - Definite diagnosis of TSC according to the Consensus criteria (Northrup,2013) - At least 1 focus of cortical dysplasia disclosed on brain MRI Are the trial subjects under 18? yes Number of subjects for this age range: 60 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: Patient may not enter the study if any of the following exclusion criteria are met: - history of seizures prior to randomization - history of antiepileptic treatment - history of treatment with mTOR inhibitor, - gestational age below 41 weeks at the day of randomization - body weight lower than 3 kg at the day of randomization - SEGA or other TSC- associated lesion requiring urgent surgical intervention - recent surgery within 1 month prior to the randomization - intercurrent infection at the date of randomization - known history of HIV seropositivity - live vaccination within 4 weeks prior to randomization - lack of first TBC and hepatitis B vaccinations - Any significant clinical, laboratory , ECG or other abnormalities, comorbidity or concomitant treatment 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. - Use of an investigational drug within 1 month prior to randomization
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of the ViRap study is to investigate safety and efficacy of rapamycin versus vigabatrin in the preventive treatment of infants with TSC. The primary epilepsy-related objective of the study is to compare the risk of developing epileptic seizures during the blinded phase of the study in the group receiving vigabatrin and the group receiving rapamycin. The primary tumor -related objective of the ViRap study is to compare the risk of new or increasing tumors in the group receiving rapamycin and the group receiving vigabatrin in the blinded phase of the study.;Secondary Objective: The secondary objective of the Virap study is to compare the safety profile of vigabatrin and rapamycin in infants with TSC and the comparison of severity of epilepsy and its neuropsychiatric comorbidities, as well as changes in the size of TSC-associated tumors in patients receiving rapamycin and vigabatrin. Additionally, the impact of treatment on the quality of life of patients receiving rapamycin or vigabatrin will be compared.;Primary end point(s): The primary endpoints of the ViRap study are: -occurrence of clinical seizures in the blinded phase of the study -increase in summarized volume of TSC-associated tumors = 25% of initial value within the blinded phase of the study.;Timepoint(s) of evaluation of this end point: The primary endpoints will be assessed at the end of the blinded phase of the study (not later than on Day730) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints include: - total volume of TSC-associated tumors within the blinded phase and the whole study - the risk for high risk of autism assessed with psychological test (ADOS) at the end of the study - the risk for low developmental quotient (< 70 points in Bayley Scales of Infant Development,) at the end of the study, - the risk of drug-resistant epilepsy at any point of the study, - occurrence of adverse events within the blinded phase of the study, - number of adverse events across the whole study - parameters of physical development (weight and height gain history) across the whole study.;Timepoint(s) of evaluation of this end point: The secondary endpoints will be analyzed respectively: - at the end of the blinded phase of the study (not later than on Day730) - at the end of the study (not later than on Day 730) - at multiple timepoints during the study. | — |
Countries
Poland
Contacts
Public ContactKatarzyna Kotulska-Józwiak
The Children's Memorial Health Institute
Outcome results
None listed