The Lancet July 16, 2022

Intravenous tenecteplase compared with alteplase for acute ischaemic stroke in Canada (AcT): a pragmatic, multicentre, open-label, registry-linked, randomised, controlled, non-inferiority trial

Menon BK, Buck BH, Singh N, et al.

Bottom Line

The AcT trial demonstrated that a single intravenous bolus of tenecteplase (0.25 mg/kg) is non-inferior to the standard one-hour infusion of alteplase (0.9 mg/kg) in achieving excellent functional outcomes for patients with acute ischemic stroke.

Key Findings

1. Excellent functional outcome (modified Rankin Scale score 0-1 at 90-120 days) was achieved in 36.9% of patients in the tenecteplase group compared to 34.8% in the alteplase group.
2. The unadjusted risk difference for the primary outcome was 2.1% (95% CI, -2.6% to 6.9%), meeting the pre-specified non-inferiority margin of -5.0%.
3. Rates of 24-hour symptomatic intracranial hemorrhage (sICH) were comparable, occurring in 3.4% of the tenecteplase arm and 3.2% of the alteplase arm.
4. Mortality at 90 days and rates of functional independence (mRS 0-2) showed no significant differences between the two thrombolytic regimens, confirming a similar overall safety and efficacy profile.

Study Design

Design
RCT, Non-inferiority
Open-Label
Sample
1,600
Patients
Duration
90-120 days
Median
Setting
Multicenter, Canada
Population Adults (≥18 years) with a disabling acute ischemic stroke presenting within 4.5 hours of symptom onset who were eligible for standard-of-care intravenous thrombolysis.
Intervention Intravenous tenecteplase (0.25 mg/kg, maximum 25 mg) administered as a single rapid bolus over 5 seconds.
Comparator Intravenous alteplase (0.9 mg/kg, maximum 90 mg) administered as a 10% initial bolus over 1 minute followed by a 1-hour continuous infusion.
Outcome Proportion of patients achieving an excellent functional outcome, defined as a modified Rankin Scale (mRS) score of 0-1 at 90-120 days.

Study Limitations

The open-label design could introduce performance or detection bias, although this was mitigated by blinded endpoint assessment.
The trial was conducted exclusively within the Canadian healthcare system, which may limit generalizability to settings with different stroke triage and transport workflows.
The pragmatic, registry-linked nature of the trial resulted in some missing secondary outcome data and variations in timing for the 90-120 day follow-up.
The majority of enrolled patients had mild-to-moderate strokes, meaning data regarding patients with extremely severe strokes at baseline may be more limited, though large vessel occlusion (LVO) subgroups behaved consistently.

Clinical Significance

The AcT trial provides definitive, large-scale evidence supporting tenecteplase as a safe and effective replacement for alteplase in acute ischemic stroke. Because tenecteplase is administered as a single 5-second bolus—rather than an initial bolus followed by a 1-hour infusion—it vastly simplifies clinical workflow. This is particularly advantageous for 'drip-and-ship' paradigms, as it eliminates the need for intravenous pumps and avoids delays or infusion interruptions during inter-facility transport for endovascular thrombectomy. These findings have catalyzed a major shift in international stroke guidelines toward tenecteplase as the preferred thrombolytic agent.

Historical Context

For more than 25 years following the landmark 1995 NINDS trial, alteplase was the only FDA-approved medical therapy for acute ischemic stroke. Tenecteplase, a genetically engineered variant of alteplase designed to have greater fibrin specificity and a longer half-life, had long superseded alteplase in the treatment of acute myocardial infarction. In stroke, earlier phase II and III trials (such as EXTEND-IA TNK and NOR-TEST) suggested tenecteplase was at least as effective, potentially yielding higher recanalization rates in large vessel occlusions. However, large, pragmatic, definitive data across all thrombolysis-eligible stroke phenotypes were needed to change widespread practice. The 2022 AcT trial served as that definitive pivot point, enrolling the largest stroke cohort for this comparison to date and firmly establishing non-inferiority.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

How does the molecular structure and pharmacokinetics of tenecteplase differ from alteplase, and why does this allow for a single-bolus administration in acute ischemic stroke?

Key Response

Tenecteplase is a genetically engineered variant of alteplase with specific amino acid substitutions that increase its half-life, resistance to plasminogen activator inhibitor-1 (PAI-1), and fibrin specificity. This longer half-life allows it to be given as a single intravenous bolus over 5 seconds rather than the one-hour continuous infusion required for alteplase.

Resident
Resident

From a practical workflow perspective in the emergency department, what are the primary advantages of utilizing tenecteplase over alteplase for a patient who is a candidate for endovascular thrombectomy and needs to be transferred to a comprehensive stroke center?

Key Response

Tenecteplase is administered as a single bolus, which eliminates the need to set up an infusion pump and monitor a continuous infusion during inter-facility transport (the 'drip and ship' model). This significantly streamlines ED workflow, reduces door-to-needle and door-to-in/out times, and simplifies the transport process for large vessel occlusion patients.

Fellow
Fellow

The AcT trial utilized a pragmatic, open-label design. How might the open-label nature of this trial influence the downstream management of patients, specifically regarding the decision to proceed with endovascular thrombectomy, and how does this affect the interpretation of the primary outcome?

Key Response

In an open-label trial, treating physicians know which thrombolytic the patient received. This knowledge could introduce performance bias, potentially altering the threshold for proceeding with rescue endovascular thrombectomy or the aggressiveness of post-thrombolytic care. Evaluating the rates of EVT between the two arms is crucial to ensure that differences in downstream management did not disproportionately drive the non-inferiority results.

Attending
Attending

Given the results of the AcT trial alongside trials like EXTEND-IA TNK, how should stroke systems of care reorganize their thrombolytic protocols, and what are the cost-effectiveness and safety implications of making a system-wide switch from alteplase to tenecteplase?

Key Response

The non-inferiority of tenecteplase, combined with its operational simplicity and lower drug cost, provides a compelling case for a system-wide switch. Attendings must consider not just the clinical non-inferiority, but the training required for nursing staff, updating pharmacy protocols, and the potential reduction in medication errors associated with avoiding complex infusion pump setups in acute, high-stress scenarios.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The AcT trial utilized a registry-linked approach and a pre-specified non-inferiority margin of -5% for the primary outcome. How does this pragmatic registry-linked data collection impact the statistical power and risk of type I error in concluding non-inferiority compared to a traditional RCT?

Key Response

Registry-linked trials are highly pragmatic and cost-effective but can suffer from missing data, loss to follow-up, or misclassification in mRS scoring by non-research clinicians. In a non-inferiority trial, misclassification and 'noise' bias the results toward the null, artificially making the two treatments look more similar and increasing the risk of falsely concluding non-inferiority (Type I error). A rigorous statistical critique must evaluate whether intention-to-treat and per-protocol analyses are concordant to mitigate this bias.

Journal Editor
Journal Editor

As a peer reviewer, how do you assess the threat of ascertainment bias in the AcT trial given its open-label design, particularly concerning secondary safety outcomes like symptomatic intracranial hemorrhage (sICH)?

Key Response

While the primary outcome (mRS at 90 days) can be partially protected via a PROBE (Prospective Randomized Open, Blinded End-point) design, acute safety outcomes like sICH might be subject to ascertainment bias if investigators look more closely for bleeding or order more imaging in one arm. An editor would flag the need for central, blinded adjudication of neuroimaging to ensure safety outcomes were assessed objectively despite the unblinded drug administration.

Guideline Committee
Guideline Committee

Based on the robust non-inferiority findings of the AcT trial and corroborating data, how should current AHA/ASA and ESO clinical practice guidelines be updated regarding the tier of recommendation for tenecteplase (0.25 mg/kg) versus alteplase for all eligible acute ischemic stroke patients?

Key Response

Historically, guidelines recommended tenecteplase primarily for patients with large vessel occlusions prior to thrombectomy (based on EXTEND-IA TNK). The AcT trial provides Class I, Level of Evidence A data supporting that tenecteplase is a reasonable alternative (non-inferior) to alteplase for all thrombolysis-eligible acute ischemic stroke patients. Guideline committees should elevate tenecteplase to a first-line agent on par with, or even preferred over, alteplase due to identical efficacy and superior logistical benefits.

Clinical Landscape

Noteworthy Related Trials

1995

NINDS Trial

n = 624 · NEJM

Tested

Alteplase 0.9 mg/kg

Population

Patients with acute ischemic stroke treated within 3 hours of symptom onset

Comparator

Placebo

Endpoint

Favorable outcome at 3 months across multiple neurologic scales

Key result: Intravenous alteplase significantly improved clinical outcomes at 3 months despite an increased risk of symptomatic intracranial hemorrhage.
2017

NOR-TEST

n = 1100 · Lancet Neurol

Tested

Tenecteplase 0.4 mg/kg

Population

Patients with acute ischemic stroke eligible for intravenous thrombolysis

Comparator

Alteplase 0.9 mg/kg

Endpoint

Excellent functional outcome (mRS 0-1) at 3 months

Key result: Tenecteplase showed similar efficacy and safety to alteplase, though the trial predominantly enrolled patients with mild stroke severity.
2018

EXTEND-IA TNK

n = 202 · NEJM

Tested

Tenecteplase 0.25 mg/kg

Population

Ischemic stroke patients with large vessel occlusion scheduled for thrombectomy

Comparator

Alteplase 0.9 mg/kg

Endpoint

Substantial reperfusion at initial angiogram

Key result: Tenecteplase resulted in a significantly higher rate of reperfusion before thrombectomy and better functional outcomes than alteplase.

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