Clinical trial · Interventional
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With 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
NCT05534672CI-TRIAL-00068673RaRE-TSrecruitingPhase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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).
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Tuberous Sclerosis Complex | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Placebo | Other | — | UNRESOLVED |
| Rapamycin | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Rapamycin arm
- description
- Each patient randomized to the rapamycin arm will receive rapamycin in liquid. The rapamycin will be administered in individually calculated doses depending on the body surface of participants
- interventionNames
- Other: Rapamycin
- type
- PLACEBO_COMPARATOR
- label
- Placebo arm
- description
- The patients assigned to the placebo arm will receive placebo in liquid, analogically to the rapamycin group.
- interventionNames
- Other: Placebo
Primary outcomes (2)
- measure
- 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
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
- Maximum age
- 50 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available
No reference posted for this study.