CFTR modulators are approved for approximately 90% of people with cystic fibrosis in the USA and provide substantial clinical benefit. N1303K (Asn1303Lys), one of the most common class 2 CFTR defects, has not been approved for these therapies by any regulatory agency. Preclinical investigation by our laboratories showed N1303K CFTR activation with elexacaftor–tezacaftor–ivacaftor (ETI). In this trial, we evaluate whether ETI improves CFTR function, measured by sweat chloride and other clinical outcomes, in people with cystic fibrosis and CFTRN1303K.

中文翻译：

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在美国囊性纤维化和 CFTRN1303K 患者中 elexacaftor-tezacaftor-ivacaftor 治疗的评估：一项前瞻性、多中心、开放标签、单臂试验


CFTR 调节剂在美国被批准用于大约 90% 的囊性纤维化患者，并提供实质性的临床益处。N1303K （Asn1303Lys） 是最常见的 2 类 CFTR 缺陷之一，尚未被任何监管机构批准用于这些疗法。我们实验室的临床前研究表明，elexacaftor-tezacaftor-ivacaftor （ETI） 可激活 N1303K CFTR。在这项试验中，我们评估了 ETI 是否改善了囊性纤维化和 CFTRN1303K 患者的 CFTR 功能，通过汗液氯化物和其他临床结果来衡量。