A multicenter, prospective, randomized, open-label, blinded endpoint trial of intravenous thrombolysis with tenecteplase for acute non-large vessel occlusion in extended time window (OPTION): Rationale and design

作者：Gaoting Ma, Ran Mo, Yingting Zuo, Shujuan Meng, Yifan Wu, Ziying Jiang, Jieying Wu, Shaoyuan Lei, Thanh N. Nguyen, Jeffrey L. Saver, Duolao Wang, Lianmei Zhong, Qingfeng Ma, Junwei Hao · 发表于：Journal of Translational Internal Medicine · 年份：2025 · DOI：10.1515/jtim-2025-0048 · 被引用次数：4 · 研究领域：Acute Ischemic Stroke Management、S100 Proteins and Annexins、Neuroinflammation and Neurodegeneration Mechanisms

Background and Objectives: Recombinant human tenecteplase (TNK) tissue-type plasminogen activator (rhTNK-tPA, also named tenecteplase) is non-inferior to alteplase when given up to 4.5 hours after acute ischemic stroke (AIS) within 4.5 hours. Whether intravenous tenecteplase compared with supportive care improves the outcome of patients with AIS due to non-large vessel occlusion (non-LVO) between 4.5-24 hours is uncertain. The Tenecteplase for Acute Non-large vessel occlusion in the Extended Time Window (OPTION) trial aims to test the efficacy and safety of intravenous tenecteplase in patients presenting between 4.5-24 hours after symptom onset who have a non-LVO and evidence of salvageable tissue on perfusion imaging. Methods: OPTION is a multicenter, prospective, randomized, open-label, blinded endpoint (PROBE) study. Patients with AIS due to non-LVO and evidence of salvageable brain tissue within 4.5-24 hour time window meeting eligibility criteria will be allocated in a 1∶1 ratio to tenecteplase or standard medical treatment. A target mismatch profile is defined as ischemic core volume <50 mL, a mismatch ratio ≥1.2, and a mismatch volume ≥10 mL. A total of 568 patients will provide >80% power to detect a 12% absolute difference in the proportion of patients achieving an excellent functional outcome (modified Rankin scale [mRS] 0-1 at 90 days) at the two-sided 0.05 significance level. The primary efficacy outcome is excellent functional outcome (mRS 0-1) at 90 days. Second...