Family Weighs 1-in-3 Placebo Risk in MAGNITUDE Trial for Hereditary Cardiac Amyloidosis
Updated
Updated · amyloidosisnewstoday.com · Aug 20
Family Weighs 1-in-3 Placebo Risk in MAGNITUDE Trial for Hereditary Cardiac Amyloidosis
2 articles · Updated · amyloidosisnewstoday.com · Aug 20
Summary
A family plans to ask the mother’s cardiologist within weeks about joining MAGNITUDE, a Phase 3 trial of Intellia’s gene-editing therapy nexiguran ziclumeran, or nex-z, for hereditary cardiac amyloidosis.
The interest centers on nex-z’s potential as a one-time treatment that stops production of the TTR protein, which misfolds and forms amyloid in ATTR cardiomyopathy.
The decision remains fraught because the FDA put the study on hold in October 2025 after a participant developed severe liver toxicity and died, though the family said newer company updates clarified which patients may face higher risk.
Eligibility and enrollment are still uncertain in Taiwan, and even if the mother qualifies, the trial carries a one-in-three chance of receiving placebo.
For patients outside the U.S., the family sees trial access as one way to avoid waiting an additional two to three years or more for approval, review and pricing of new therapies.