Heart failure & cardiomyopathiesRandomised controlled trial
Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy
- Randomised controlled trial
- phase 3
- double-blind
- placebo-controlled
- 2:1 randomisation
Authors' conclusion
In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome containing components of mortality, morbidity, and function than placebo. Adverse events were similar in the two groups.
Study context
- Population
- Patients with transthyretin amyloid cardiomyopathy
- Participants
- 632 patients
- Comparison
- Acoramidis vs placebo
Primary result
As reported in the abstract
Win ratio
1.8
95% CI 1.4–2.2
P < 0.001
Acoramidis vs Placebo
win ratio 1.8 (95% CI 1.4–2.2); P < 0.001
- All-cause death and CV-related hospitalization contributed >half the wins and losses (% of all pairwise comparisons)
- 58%
- NT-proBNP pairwise comparisons: wins vs losses (highest ratio)
- 23.3% vs. 7.0%
Safety
Any adverse event · similar in the two groups
Serious adverse events
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy. N Engl J Med. Published 11 January 2024. doi:10.1056/nejmoa2305434
Summarised from the published abstract; every number is checked against it