tuberous sclerosis complex, epilepsy, organ tumors associated with tuberous sclerosis
Conditions
Interventions
Trade Name: Rapamune 1 mg/ml oral solution
Pharmaceutical Form: Oral solution
Pharmaceutical form of the placebo: Oral liquid
Route of administration of the placebo: Oral use
Sponsors
The Children's Memorial Health Institute
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -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
Exclusion criteria
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).;Secondary Objective: The secondary aim of the study is to ascertain the impact of rapamycin on the TSC- Associated Neuropsychiatric Disorders (TAND) and TSC-associated tumors (brain, kidney, retina and skin lesions) in comparison to placebo. ;Primary end point(s): - comparison of the number of patients with at least 50% reduction of seizures per week in the last month of the core blinded phase in comparison to screening phase in the rapamycin vs placebo group - number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase;Timepoint(s) of evaluation of this end point: -interim analysis -final analyses after the formal final database lock, planned within one month after the last patient last visit in the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -comparison of the number of seizures per week and the number of days free of seizures in the rapamycin vs placebo group, during 12-week treatment in double-blind core phase - severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group;Timepoint(s) of evaluation of this end point: -interim analysis -final analyses after the formal final database lock, planned within one month after the last patient last visit in the study | — |
Countries
Poland
Contacts
Public ContactKatarzyna Kotulska-Józwiak
The Children's Memorial Health Institute
Outcome results
None listed