SIROLIMUS for Tuberous sclerosis complex|Drug-resistant epilepsy|TSC-associated tumors

Inclusion criteria

• {"criterion_text":"- Parents/caregivers are willing to and able to give informed consent form for the participation in the study"}
• {"criterion_text":"- Parents/caregivers are willing to and able to comply with all study requirements"}
• {"criterion_text":"- Definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)"}
• {"criterion_text":"- drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks"}
• {"criterion_text":"- male or female aged from 3 months up to 55 years at the day of randomization"}
• {"criterion_text":"- body weight of at least 6 kg and proper nutritional status (assessed by investigator)"}

Exclusion criteria

• {"criterion_text":"- history of treatment with mTOR inhibitor in the three months prior to screening"}
• {"criterion_text":"- history of pseudo-epileptic seizures"}
• {"criterion_text":"- history of progressive CNS disease other than TSC"}
• {"criterion_text":"- recent surgery within 2 weeks prior to the screening"}
• {"criterion_text":"- severe infection within 2 weeks prior to the screening"}
• {"criterion_text":"- contraindications for MRI or general anesthesia"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- pregnancy"}
• {"criterion_text":"- hypersensitivity to the active substance or to any of the excipients of IMP / placebo"}

Primary endpoints

• {"endpoint_text":"- 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 (","definition_or_measurement_approach":"Proportion of patients achieving ≥50% reduction in weekly seizure frequency comparing the last month of the core blinded phase to the screening phase (seizure counts per week)."}
• {"endpoint_text":"- number of adverse events (according to CTCAE classification) in the rapamycin vs placebo group during the double-blind core phase","definition_or_measurement_approach":"Count and classification of adverse events using CTCAE criteria during the double-blind core phase, compared between rapamycin and placebo groups."}

Secondary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Weekly seizure frequency and seizure-free days during the 12-week double-blind core treatment period compared between rapamycin and placebo arms."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Assessment of AE severity per CTCAE and count of participants withdrawn due to AEs, compared between treatment groups."}

Poland

Earliest CTIS Part Ii Submission Date

29-10-2024

Latest Decision Or Authorization Date

09-03-2026

Processing Time Days

496

Number Of Sites

4

Number Of Participants

200

Primary sponsor

Full Name

Instytut Pomnik Centrum Zdrowia Dziecka

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Poland

Third parties

• {"country":"Poland","full_name":"Uniwersytet Medyczny W Lodzi","duties_or_roles":"genetic testing, biomarkers analysis","organisation_type":"Educational Institution"}
• {"country":"Poland","full_name":"Transition Technologies Science","duties_or_roles":"sponsorDuties codes: 10; 6","organisation_type":"Industry"}
• {"country":"Poland","full_name":"Instytut Pomnik Centrum Zdrowia Dziecka","duties_or_roles":"PK testing, EEG, imaging diagnostics","organisation_type":"Hospital/Clinic/Other health care facility"}