Evaluating Rapamycin for Drug Resistant Epilepsy in Tuberous Sclerosis Complex
Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With TSC
This study is testing if rapamycin can help people with drug-resistant epilepsy due to tuberous sclerosis complex feel better compared to a placebo.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 200 (estimated) |
| Ages | 3 Months to 50 Years |
| Sex | All |
| Sponsor | Children's Memorial Health Institute, Poland Academic / other |
| Locations | 2 sites (Lodz and 1 other locations) |
| Trial ID | NCT05534672 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, and efficacy of rapamycin compared to a placebo in patients with drug-resistant epilepsy associated with tuberous sclerosis complex (TSC). It is a randomized, double-blind, placebo-controlled trial that includes three phases: screening, dose adjustment, and a core blinded phase, followed by an open-label observation. Eligible participants will be randomized in a 1:1 ratio to receive either rapamycin or placebo, with stratification based on age, sex, and prior antiepileptic drug use.
Who should consider this trial
Good fit: Ideal candidates are males and females aged 3 months to 50 years with a confirmed diagnosis of TSC and experiencing drug-resistant epilepsy.
Not a fit: Patients with a history of treatment with mTOR inhibitors or those with other significant comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients suffering from drug-resistant epilepsy associated with TSC.
How similar studies have performed: While this approach is novel in the context of TSC-related epilepsy, previous studies have shown promise in using rapamycin for other conditions, suggesting potential for success.
Eligibility criteria
Show full inclusion / exclusion 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 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 this trial is running
Lodz and 1 other locations
- Medical University of Lodz — Lodz, Poland (Not_yet_recruiting)
- Children's Memorial Health Institute, Neurology and Epileptology — Warsaw, Poland (Recruiting)
Study contacts
- Study coordinator: Katarzyna Kotulska-Jozwiak
- Email: k.kotulska@ipczd.pl
- Phone: +48 22 8157404
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.