Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
이 임상시험이 검증하는 내용
This is the study that made Casgevy an approved medicine. Participants with severe sickle cell disease had their own blood stem cells edited and returned. The main question was whether they would stop having the severe pain crises that define the disease — and for the large majority, they did.
An open-label, single-arm, multi-site Phase 2/3 study of autologous CRISPR-Cas9-modified CD34+ cells (CTX001, later exagamglogene autotemcel) in patients aged 12–35 with severe sickle cell disease. The primary endpoint was freedom from severe vaso-occlusive crises for at least 12 consecutive months. Results supported approvals by the MHRA, FDA and EMA.
Sources
- New England Journal of Medicine · 2024
Exagamglogene autotemcel for severe sickle cell disease ↗
임상시험 결과가 의미하는 것과 의미하지 않는 것
초기 임상 결과는 소수의 참가자와 짧은 추적 관찰 기간에서 얻은 것입니다. 참가자 수와 시간이 늘어나면 결과가 달라질 수 있으며, 모집 중·진행 중·완료된 임상시험이라도 치료 효과가 입증된 것은 아닙니다. 등록 기준은 엄격하며, 참여 자격은 임상시험 기관이 결정하고 웹사이트가 결정하지 않습니다.