Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
本试验正在测试什么
A large late-stage trial testing whether a single infusion of a CRISPR treatment, which switches off a gene in the liver, helps people whose hearts are being damaged by amyloid deposits. In 2025 a participant died of liver failure after dosing; the trial was paused and placed on hold by the FDA, and the hold was later lifted.
A global Phase 3 study of nexiguran ziclumeran, a lipid-nanoparticle CRISPR-Cas9 therapy knocking out hepatic TTR, in participants with ATTR cardiomyopathy. Following a serious hepatic adverse event and death in a participant in his early eighties, Intellia voluntarily paused dosing and screening; the FDA imposed a clinical hold, which was subsequently lifted for this study.
Sources
- Intellia Therapeutics · 2025
Intellia provides update on MAGNITUDE clinical trials ↗ - Intellia Therapeutics · 2025
Intellia announces FDA lift of clinical hold on MAGNITUDE Phase 3 clinical trial in ATTR-CM ↗
试验结果意味着什么，又不意味着什么
早期试验结果来自少量参与者和较短的随访周期，随着人数增加和时间延长，结论可能发生变化。正在招募、进行中甚至已完成的试验，并不代表某种疗法已被证实有效。入组标准十分严格，是否符合条件由试验机构决定，而非由网站判断。