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
The earlier trial of the hereditary angioedema treatment, testing whether a single infusion could reduce swelling attacks. Its results were published in a major medical journal and supported the move into the Phase 3 study that followed.
A Phase 1/2 study of NTLA-2002 in adults with hereditary angioedema, evaluating safety, pharmacodynamics and attack rate following a single dose. Results were published in the New England Journal of Medicine and supported progression to the Phase 3 HAELO study.
Sources
- New England Journal of Medicine · 2024
CRISPR-based therapy for hereditary angioedema ↗
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。