Division of Pulmonary and Critical Care Medicine, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA (A.B.W., D.M.S.).
Acceleron Pharma, a wholly owned subsidiary of Merck & Co Inc, Rahway, NJ (S.M.).
Circ Heart Fail. 2024 May;17(5):e011227. doi: 10.1161/CIRCHEARTFAILURE.123.011227. Epub 2024 Apr 4.
This study aims to assess the impact of sotatercept on exercise tolerance, exercise capacity, and right ventricular function in pulmonary arterial hypertension.
SPECTRA (Sotatercept Phase 2 Exploratory Clinical Trial in PAH) was a phase 2a, single-arm, open-label, multicenter exploratory study that evaluated the effects of sotatercept by invasive cardiopulmonary exercise testing in participants with pulmonary arterial hypertension and World Health Organization functional class III on combination background therapy. The primary end point was the change in peak oxygen uptake from baseline to week 24. Cardiac magnetic resonance imaging was performed to assess right ventricular function.
Among the 21 participants completing 24 weeks of treatment, there was a significant improvement from baseline in peak oxygen uptake, with a mean change of 102.74 mL/min ([95% CIs, 27.72-177.76]; =0.0097). Sotatercept demonstrated improvements in secondary end points, including resting and peak exercise hemodynamics, and 6-minute walk distance versus baseline measures. Cardiac magnetic resonance imaging showed improvements from baseline at week 24 in right ventricular function.
The clinical efficacy and safety of sotatercept demonstrated in the SPECTRA study emphasize the potential of this therapy as a new treatment option for patients with pulmonary arterial hypertension. Improvements in right ventricular structure and function underscore the potential for sotatercept as a disease-modifying agent with reverse-remodeling capabilities.
URL: https://www.clinicaltrials.gov; Unique identifier: NCT03738150.
本研究旨在评估 sotatercept 对肺动脉高压患者的运动耐量、运动能力和右心功能的影响。
SPECTRA（肺动脉高压中 sotatercept 的 2 期探索性临床试验）是一项 2 期 a 期、单臂、开放标签、多中心探索性研究，评估了 sotatercept 在背景联合治疗下对肺动脉高压和世界卫生组织功能分类 III 级患者的侵入性心肺运动测试中的影响。主要终点是从基线到第 24 周时峰值摄氧量的变化。进行心脏磁共振成像以评估右心功能。
在完成 24 周治疗的 21 名参与者中，与基线相比，峰值摄氧量有显著改善，平均变化为 102.74 mL/min（[95%CI，27.72-177.76]；=0.0097）。sotatercept 在次要终点方面表现出改善，包括静息和峰值运动血流动力学以及 6 分钟步行距离与基线测量值相比。心脏磁共振成像显示在第 24 周时右心功能较基线有所改善。
SPECTRA 研究中 sotatercept 的临床疗效和安全性强调了这种治疗方法作为肺动脉高压患者新治疗选择的潜力。右心结构和功能的改善强调了 sotatercept 作为一种具有反向重塑能力的疾病修饰药物的潜力。
