Heart failure & cardiomyopathiesRandomised controlled trial
Eplontersen for Transthyretin Amyloid Cardiomyopathy
- Randomised controlled trial
- phase 3
- international
- double-blind
- placebo-controlled
Authors' conclusion
Among patients with ATTR-CM, eplontersen therapy did not lead to a lower risk of a composite of death from cardiovascular causes and recurrent cardiovascular events than placebo up to 140 weeks.
Study context
- Population
- Patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)received ≥1 dose
- Participants
- 1,432 patients
- Comparison
- Eplontersen vs placebo
- Eplontersenn = 715
- Placebon = 717reference
- Primary endpoint
- Cumulative composite of:
- CV death
- Recurrent CV clinical events
- Follow-up
- Up to week 140
Primary result
As reported in the abstract
Rate ratio
0.89
95% CI 0.73–1.09
P = 0.28
Events by treatment group
Eplontersen vs Placebo
rate ratio 0.89 (95% CI 0.73–1.09); P = 0.28
Further findings
| Outcome | rate ratio (95% CI) | |
|---|---|---|
| Composite of all-cause death and CV events · up to week 140 | 0.86 (0.71–1.04) | |
| 0.512Rate ratio (log scale) |
events
| Outcome | Eplontersen | Placebo | Estimate |
|---|---|---|---|
| CV death (component of primary end point) | 74 | 70 | |
| CV clinical events (component of primary end point) | 307 | 322 |
Safety
Serious adverse events
Original publication
Eplontersen for Transthyretin Amyloid Cardiomyopathy. N Engl J Med. Published 28 August 2026. doi:10.1056/nejmoa2608510
Summarised from the published abstract; every number is checked against it