Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Epilepsy: 3 are currently recruiting; 1 is active or not yet recruiting; 4 have completed. The most advanced is Phase 3 (NCT05534672). It plans or enrolled 200 participants. Registration activity spans 2006 to 2026. The literature layer holds 5 publications for this pair: 2 Cochrane reviews and 3 clinical trial publications. Publication years run from 2014 to 2024. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Epilepsy ranks 187 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: 60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by syndrome and duration of freedom).
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 64.0 (trials 28.0, literature 16.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 3 recruiting, 1 active / not yet recruiting, 4 completed, 0 other |
| Linked publications | 5 (3 clinical trial publications, 2 Cochrane reviews) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | AARS1 |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT05534672 | Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex 2023 | recruiting | Phase 3 | 200 |
| NCT03198949 | A Study Investigating the Anti-epileptic Efficacy of Afinitor (Everolimus) in Patients With Refractory Seizures Who Have Focal Cortical Dysplasia Type II (FCD II) 2018 | completed | Phase 2 | 23 |
| NCT02451696 | A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD 2014 | completed | Phase 2 | 15 |
| NCT07680322 | A Phase 2 Study of the Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy 2026 | not yet recruiting | Phase 2 | 42 |
| NCT05104983 | Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study 2021 | recruiting | Phase 2 | 64 |
| NCT00509431 | Erlotinib and Sirolimus in Treating Patients With Recurrent Malignant Glioma 2006 | completed | Phase 1 | 19 |
| NCT07095933 | The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy 2026 | recruiting | Early Phase 1 | 5 |
| NCT04595513 | Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants 2020 | completed | PHASE1, PHASE2 | 5 |
Published literature
- Cochrane review Prenatal mTOR Inhibitors in Tuberous Sclerosis Complex: Current Insights and Future DirectionsRacioppi G, Proietti Checchi M, Sforza G et al. · Journal of clinical medicine · 2024 · PMID 39518472
- Clinical trial publication Effectiveness and safety of everolimus treatment in patients with tuberous sclerosis complex in real-world clinical practiceCockerell I, Christensen J, Hoei-Hansen CE et al. · Orphanet journal of rare diseases · 2023 · PMID 38042867
- Cochrane review Interaction of cannabidiol with other antiseizure medications: A narrative reviewGilmartin CGS, Dowd Z, Parker APJ et al. · Seizure · 2021 · PMID 33541771
- Clinical trial publication Sirolimus for epilepsy in children with tuberous sclerosis complex: A randomized controlled trialOverwater IE, Rietman AB, Bindels-de Heus K et al. · Neurology · 2016 · PMID 27511181
- Clinical trial publication Mammalian target of rapamycin inhibitors for intractable epilepsy and subependymal giant cell astrocytomas in tuberous sclerosis complexCardamone M, Flanagan D, Mowat D et al. · The Journal of pediatrics · 2014 · PMID 24518170
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target AARS1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Epilepsy?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Epilepsy?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Epilepsy: 3 are currently recruiting; 1 is active or not yet recruiting; 4 have completed. The most advanced is Phase 3 (NCT05534672). It plans or enrolled 200 participants. Registration activity spans 2006 to 2026.
What does the evidence show for Sirolimus in Epilepsy?
Sirolimus has both completed registered trials and synthesis-level publications linked to Epilepsy. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL, in this case via the biomarker or target AARS1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.