Summary
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Amyotrophic Lateral Sclerosis (ALS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Amyotrophic Lateral Sclerosis (ALS). ClinicalTrials.gov lists 1 registered trial linking Sirolimus to Amyotrophic Lateral Sclerosis (ALS): 1 has completed. The most advanced is Phase 2 (NCT03359538). It plans or enrolled 63 participants. Registered activity dates to 2017. No published literature item is linked to Sirolimus and Amyotrophic Lateral Sclerosis (ALS) 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 Amyotrophic Lateral Sclerosis ranks 50 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: Essentially zero — uniformly progressive, fatal within 2-5 years median; <10% survive >10 years; spontaneous prolonged stabilisation in <1% (SOD1 heterozygous late-onset).
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 23.5 (trials 3.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 0 recruiting, 0 active / not yet recruiting, 1 completed, 0 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 | AGTPBP1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT03359538 | Rapamycin Treatment for ALS 2017 | completed | Phase 2 | 63 |
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 AGTPBP1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Sirolimus approved for Amyotrophic Lateral Sclerosis (ALS)?
Sirolimus has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Amyotrophic Lateral Sclerosis (ALS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Amyotrophic Lateral Sclerosis (ALS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Sirolimus in clinical trials for Amyotrophic Lateral Sclerosis (ALS)?
ClinicalTrials.gov lists 1 registered trial linking Sirolimus to Amyotrophic Lateral Sclerosis (ALS): 1 has completed. The most advanced is Phase 2 (NCT03359538). It plans or enrolled 63 participants. Registered activity dates to 2017.
What does the evidence show for Sirolimus in Amyotrophic Lateral Sclerosis (ALS)?
Sirolimus has 1 completed registered trial for Amyotrophic Lateral Sclerosis (ALS) 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 AGTPBP1. 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 2026-07-06. Page built 2026-10-07.