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
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 ↗
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.