Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Multiple Sclerosis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Multiple Sclerosis. ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Multiple Sclerosis: 1 is active or not yet recruiting; 6 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03525834). The largest enrolment is 52 participants (NCT03140449). Registration activity spans 2002 to 2021. No published literature item is linked to Sirolimus and Multiple Sclerosis in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Multiple Sclerosis ranks 371 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: Untreated relapsing MS: ~1 relapse/year; disability accumulates over time in ~85% who convert to secondary progressive MS; spontaneous remission from MS is not possible.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 47.0 (trials 27.0, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 1 active / not yet recruiting, 6 completed, 1 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ATP6V1B2 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT03525834 | Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex. 2018 | completed | Phase 4 | 40 |
| NCT03140449 | Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis 2013 | completed | Phase 3 | 52 |
| NCT02201212 | Everolimus for Cancer With TSC1 or TSC2 Mutation 2014 | completed | Phase 2 | 30 |
| NCT00126672 | RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS 2005 | completed | Phase 2 | 36 |
| NCT00457808 | Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM 2002 | completed | Phase 2 | 25 |
| NCT01954693 | A Study of Everolimus in the Treatment of Neurocognitive Problems in Tuberous Sclerosis 2012 | status unknown | Phase 2 | 48 |
| NCT05467397 | Feasibility of [11C]Acetate-PET in LAM and TSC 2021 | active, not recruiting | PHASE1, PHASE2 | 7 |
| NCT04595513 | Stopping TSC Onset and Progression 2: Epilepsy Prevention in TSC Infants 2020 | completed | PHASE1, PHASE2 | 5 |
Published literature
No publication is linked to this pair yet.
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 ATP6V1B2. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Multiple Sclerosis?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Multiple Sclerosis would be drug repurposing rather than first-in-human development. This does not mean it is approved for Multiple Sclerosis. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Multiple Sclerosis?
ClinicalTrials.gov lists 8 registered trials linking Sirolimus to Multiple Sclerosis: 1 is active or not yet recruiting; 6 have completed; 1 is terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT03525834). The largest enrolment is 52 participants (NCT03140449). Registration activity spans 2002 to 2021.
What does the evidence show for Sirolimus in Multiple Sclerosis?
Sirolimus has 6 completed registered trials for Multiple Sclerosis but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. 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 ATP6V1B2. 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.