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
The companion study to the sickle cell trial, in people with beta thalassemia who needed regular blood transfusions to live. The question was whether editing their own stem cells would let them stop. Most participants became transfusion-independent.
An open-label, single-arm Phase 2/3 study of exagamglogene autotemcel in patients aged 12–35 with transfusion-dependent beta thalassemia. The primary endpoint was transfusion independence, defined as maintaining a weighted average haemoglobin of at least 9 g/dL without transfusion for at least 12 consecutive months.
Sources
- New England Journal of Medicine · 2024
Exagamglogene autotemcel for transfusion-dependent β-thalassemia ↗
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.