Summary
Alteplase has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Stroke (Ischaemic / Cerebrovascular Disease) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Stroke (Ischaemic / Cerebrovascular Disease). ClinicalTrials.gov lists 8 registered trials linking Alteplase to Stroke (Ischaemic / Cerebrovascular Disease): 3 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05626972). The largest enrolment is 113035 participants (NCT05395351). Registration activity spans 2011 to 2023. The literature layer holds 12 publications for this pair: 5 Cochrane reviews, 1 meta-analysis, 1 systematic review and 5 clinical trial publications. The dated items are from 2026. 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 Ischemic Stroke ranks 577 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: Spontaneous early neurological improvement in 10-20% within 24 hours of ischaemic stroke without intervention; functional independence at 3 months in only 40-50% without revascularisation; 15% 30-day mortality untreated.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 64.5 (trials 14.5, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 3 completed, 5 other |
| Linked publications | 12 (5 Cochrane reviews, 5 clinical trial publications, 1 meta-analysis, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT05626972 | Tenecteplase Compared to Alteplase for Patients With Large Vesel Oclusion Suspicion Before Thrombectomy 2022 | status unknown | Phase 3 | 500 |
| NCT01220739 | Safety of Rt-PA + Transcranial Emission of Low-Energy Lasers for Acute Stroke Recovery 2011 | terminated | Phase 2 | 12 |
| NCT05395338 | 1-year Clinical Outcomes in Recombinant Tissue Plasminogen Activator (Rt-PA) Treated Chinese Acute Ischaemic Stroke (AIS) Patients 2022 | completed | Not applicable | 12,551 |
| NCT05395351 | Alteplase in Elderly Acute Ischemic Stroke (AIS) Patients During Hospitalization 2022 | completed | Not applicable | 113,035 |
| NCT06018181 | Outcomes After Intravenous Alteplase / Tenecteplase With or Without Shuxuetong Injection in Routine Clinical Practice 2023 | completed | Not applicable | 2,408 |
| NCT05644938 | Optimizing Door-to-reperfusion Times of One-stop Management in Acute Ischemic Stroke 2023 | status unknown | Not applicable | 50 |
| NCT03392792 | Regisry of the Stroke Patients in Assiut University Hospital 2018 | status unknown | Not applicable | 1 |
| NCT03997292 | Intravenous Thrombolysis Registry for Acute Ischemic Stroke in China 2018 | withdrawn | Not applicable | — |
Published literature
- Clinical trial publication Predictors and Consequences of Serious Adverse Events in Patients Treated With Thrombolysis: A Prespecified Secondary Analysis of the AcT TrialAppoo A, Ignacio K, Doolan C et al. · Journal of the American Heart Association · 2026 · PMID 42261967
- Clinical trial publication Association of 24-Hour Computed Tomography Infarct Density on Functional Outcomes in Stroke: Secondary Analysis From the AcT TrialPensato U, Zhang J, Barakhanov K et al. · Journal of the American Heart Association · 2026 · PMID 42179273
- Clinical trial publication Effect of time on reperfusion in acute ischemic stroke treated with thrombolysis within the 4.5-hour window: a secondary analysis of the AcT TrialIgnacio KHD, Pensato U, Bala F et al. · Journal of stroke and cerebrovascular diseases : the official journal of National Stroke Association · 2026 · PMID 42173266
- Clinical trial publication Adjunctive Intra-Arterial Alteplase After Successful Thrombectomy for Acute Ischemic Stroke: The CHOICE-2 Randomized Clinical TrialRenú A, Fernández-Couto MD, de la Riva P et al. · JAMA · 2026 · PMID 42096239
- Clinical trial publication Efficacy and Safety of Reteplase Versus Alteplase in Acute Ischemic Stroke Based on Fibrinogen Levels: The RAISE Trial SubgroupWang J, He D, Gu H et al. · Journal of the American Heart Association · 2026 · PMID 42089199
- Cochrane review Comparative efficacy and safety between intravenous recombinant human prourokinase and alteplase in acute ischemic stroke treatment: a meta-analysis of randomized controlled trialsMaximiano ML, Gonçalves OR, Falcão L et al. · Minerva medica · 2026 · PMID 42312728
- Cochrane review Relationship between blood pressure level with prognosis of acute ischemic stroke patients treated with intravenous thrombolysis: a systematic review and meta-analysisLi Y, Huang G, Li S et al. · BMC neurology · 2026 · PMID 42106636
- Cochrane review Safety and Efficacy of Thrombolysis Prior to Thrombectomy in Large-Vessel Acute Ischemic Stroke: An Updated Systematic Review and Meta-AnalysisMohammed O, Thomas S, Velamparambil Sunny S et al. · The American journal of cardiology · 2026 · PMID 42055181
- Cochrane review Intravenous thrombolysis versus standard medical therapy in minor stroke patients with large vessel severe stenosis or occlusion: a systematic review and meta-analysisZhang T, Tong L, Xu L et al. · BMC medicine · 2026 · PMID 41792788
- Cochrane review Tenecteplase versus alteplase for acute ischemic strokePrabhakar H, Kapoor I, Mahajan C et al. · The Cochrane database of systematic reviews · 2026 · PMID 41609012
- Meta-analysis Cost-Effectiveness of Thrombectomy With or Without Alteplase in Large Vessel Occlusion Stroke: A Meta-Analysis Considering Time-to-TreatmentNguyen CP, Cavalcante F, van der Zee DJ et al. · Neurology · 2026 · PMID 41996656
- Systematic review Efficacy, safety and dosing of intravenous alteplase administered beyond 4.5 hours for ischemic stroke: systematic review and meta-analysisAlrabadi B, Alghzawi Y, Mater HI et al. · The International journal of neuroscience · 2026 · PMID 41770526
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. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Alteplase approved for Stroke (Ischaemic / Cerebrovascular Disease)?
Alteplase has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Stroke (Ischaemic / Cerebrovascular Disease) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Stroke (Ischaemic / Cerebrovascular Disease). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Alteplase in clinical trials for Stroke (Ischaemic / Cerebrovascular Disease)?
ClinicalTrials.gov lists 8 registered trials linking Alteplase to Stroke (Ischaemic / Cerebrovascular Disease): 3 have completed; 5 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT05626972). The largest enrolment is 113035 participants (NCT05395351). Registration activity spans 2011 to 2023.
What does the evidence show for Alteplase in Stroke (Ischaemic / Cerebrovascular Disease)?
Alteplase has both completed registered trials and synthesis-level publications linked to Stroke (Ischaemic / Cerebrovascular Disease). 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. 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.