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Research summary · AI summary · not yet reviewedHuman studyPhase 3Trial registry record – peer review not applicable
Phase 3 Study on Rapamycin for Drug-Resistant Epilepsy in Patients with Tuberous Sclerosis Complex
A Phase 3 interventional study planned to enroll 200 patients aged 3 months to 50 years with drug-resistant epilepsy related to Tuberous Sclerosis Complex (TSC). The study aimed to compare seizure reduction between rapamycin and placebo groups, as well as assess adverse events. However, no results have been published yet.
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This plain-English summary was written by AI from a trial registry record and may contain errors. It is not medical advice. Read the original study and talk to your MS team before making decisions about treatment.
- Human efficacy
- Not yet shown in people
- Mechanism
- Not reported
- Evidence strength
- No results yet
- Outcome
- Not reported
Why it matters
This research seeks to determine if rapamycin can effectively reduce seizures in patients with TSC, a condition with significant unmet medical needs. The outcomes could lead to new treatment avenues for drug-resistant epilepsy.
What this does not prove
No results are posted; therefore, efficacy and safety outcomes have not been established, leaving the study's impact unknown.
Next milestone
No next milestone was established from the available source.
Study facts
- Study design
- Interventional
- Participants / samples
- 200 · estimated
- Randomised
- Yes
- Controlled
- Yes
- Primary endpoint met
- Not reported
- Relevant MS type
- Not reported
- Publication date
- Not reported
- Evidence reviewed
- Trial registry record
- Regulatory approval
- Not reported
- Research areas
- Not classified
- Biological target
- Not reported
- Pathway
- Not reported
- Population
- male or female aged from 3 months up to 50 years with definite diagnosis of Tuberous Sclerosis Complex and drug-resistant epilepsy associated with TSC
- Size of effect
- Not reported
- Secondary endpoints
- 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treatment in double-blind core phase; severity of adverse events (according to CTCAE) and the number of patients withdrawn from the study due to adverse events in the rapamycin vs placebo group
- MRI endpoints
- Not reported
- Biomarkers
- Not reported
- Safety
- Not reported
Original sources
Supporting passages (19)
study phasePhase: Phase 3
study designStudy type: Interventional
subjectsParticipants: people; ages from 3 Months to 50 Years; sex All
speciesParticipants: people; ages from 3 Months to 50 Years; sex All
sample sizeEnrollment: 200 participants (estimated)
sample size basisEnrollment: 200 participants (estimated)
randomizedAllocation: Randomized
controlledArm types: Experimental; Placebo comparator
peer reviewedPublication date: unknown.
interventionInterventions: Rapamycin; Placebo
comparatorInterventions: Rapamycin; Placebo
primary endpointPrimary endpoints: 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
follow upFollow-up: final analyses after the formal final database lock, planned within one month after the last patient last visit in the study
population"eligibilityCriteria": "Inclusion Criteria:\n\n* male or female aged from 3 months up to 50 years at the day of randomization\n* patients/parents/caregivers are willing to and able to give informed consent form for the participation in the study\n* patients/parents/caregivers are willing to and able to comply with all study requirements\n* definite diagnosis of TSC according to the Consensus criteria (Northrup, 2013)\n* drug-resistant epilepsy associated with TSC with at least 8 seizures during 4 weeks\n\nExclusion Criteria:\n\n* history of treatment with mTOR inhibitor in the three months prior to screening,\n* history of pseudo-epileptic seizures,\n* history of progressive CNS disease other than TSC\n* recent surgery within 2 weeks prior to the screening\n* severe infection within 2 weeks prior to the screening\n* use of the cannabis derivatives\n* contraindications for MRI or general anesthesia\n* 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\n* pregnancy",
secondary endpoints"measure": "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",
human efficacyNo results are posted.
findingsTrial registry record. Publication date: unknown. No results are posted. Planned enrolment and endpoints are not findings.
limitationsTrial registry record. Publication date: unknown. No results are posted. Planned enrolment and endpoints are not findings.
peer reviewedTrial registry record.
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