Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
Recruiting · NCT05534672 · Interventional (participants receive a specific treatment) · Lead sponsor: Katarzyna Kotulska
View the official record on ClinicalTrials.gov →Interventions studied
PlaceboRapamycin
What this trial is about
The purpose 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).
Who can take part
Age range
3 Months to 50 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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
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
Where it is running
2 locations listed.
- Medical University of Lodz - Lodz, Poland
- Children's Memorial Health Institute, Neurology and Epileptology - Warsaw, Poland