Department of Neurology, Kaohsiung Medical University Hospital, Kaohsiung, Taiwan.
College of Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan.
Drug Des Devel Ther. 2024 Jun 6;18:2033-2042. doi: 10.2147/DDDT.S460831. eCollection 2024.
Odatroltide (LT3001), a novel small synthetic peptide molecule designed to recanalize occluded blood vessels and reduce reperfusion injury, is safe and efficacious in multiple embolic stroke animal models. This study aimed to investigate the safety and tolerability of intravenous administration of odatroltide in patients with acute ischemic stroke within 24 hours of onset.
Patients with National Institutes of Health Stroke Scale (NIHSS 4-30) who were untreated with intravenous thrombolysis or endovascular thrombectomy were randomized (2:1) to receive a single dose of odatroltide (0.025 mg/kg) or placebo within 24 hours of stroke symptom onset. The primary safety outcome was symptomatic intracranial hemorrhage (sICH) occurrence within 36 hours.
Twenty-four patients were enrolled and randomized; of these 16 and 8 received intravenous odatroltide infusion and placebo, respectively. sICH did not occur in both groups, and other safety measures were comparable between the groups. The rate of excellent functional outcome (modified Rankin Scale score, 0-1, at 90 days) was 21% and 14% in the odatroltide and placebo groups, respectively. Furthermore, 47% and 14% of patients in the odatroltide and placebo groups, respectively, showed major neurological improvement (NIHSS improvement ≥4 points from baseline to 30 days). Among the 9 odatroltide-treated patients with baseline NIHSS ≥6, 78% showed major neurological improvement.
Compared with placebo, treatment with intravenous odatroltide within 24 hours following onset of ischemic stroke appears to be safe and may be associated with better neurological and functional outcomes. However, the efficacy and safety of odatroltide requires further confirmation in the next phase of clinical trials.
Clinicaltrials.gov identifier: NCT04091945.
Odatroltide（LT3001）是一种新型的小合成肽分子，旨在使闭塞的血管再通并减少再灌注损伤，在多种栓塞性卒中动物模型中具有安全性和疗效。本研究旨在探讨在发病 24 小时内接受急性缺血性卒中治疗的患者静脉注射 odatroltide 的安全性和耐受性。
未接受静脉溶栓或血管内取栓治疗的 NIHSS 评分（4-30）的患者，在发病后 24 小时内随机（2:1）接受单次剂量的 odatroltide（0.025mg/kg）或安慰剂治疗。主要安全性结局是 36 小时内发生症状性颅内出血（sICH）。
24 例患者入选并随机分组，其中 16 例和 8 例患者分别接受了 odatroltide 静脉输注和安慰剂治疗。两组均未发生 sICH，且两组间其他安全性措施无差异。在 odatroltide 和安慰剂组中，90 天时改良 Rankin 量表评分（0-1 分）的优良功能结局发生率分别为 21%和 14%。此外，odatroltide 和安慰剂组中分别有 47%和 14%的患者在 30 天时出现主要神经功能改善（NIHSS 较基线改善≥4 分）。在基线 NIHSS 评分≥6 的 9 例 odatroltide 治疗患者中，78%的患者出现了主要神经功能改善。
与安慰剂相比，在缺血性卒中发病后 24 小时内静脉使用 odatroltide 似乎是安全的，并且可能与更好的神经和功能结局相关。然而，odatroltide 的疗效和安全性需要进一步在临床试验的下一阶段得到证实。
Clinicaltrials.gov 标识符：NCT04091945。
