Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
Qué está evaluando este ensayo
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 ↗
Qué significa y qué no significa el resultado de un ensayo
Los resultados de fases tempranas provienen de un número reducido de participantes y un seguimiento breve. Pueden cambiar con más personas y más tiempo, y un ensayo en reclutamiento, activo o incluso completado no ha demostrado que un tratamiento funcione. Los criterios de inscripción son estrictos; la elegibilidad la decide el centro del ensayo, nunca un sitio web.