Prevention & lipidsRandomised controlled trial
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
- Randomised controlled trial
- phase 3
- double-blind
Authors' conclusion
Among adults with heterozygous familial hypercholesterolemia, those who received inclisiran had significantly lower levels of LDL cholesterol than those who received placebo, with an infrequent dosing regimen and an acceptable safety profile.
Study context
- Population
- Adults with heterozygous familial hypercholesterolemia
- Participants
- 482 adults
- Comparison
- Inclisiran vs placebo
- Primary endpoint
- Percent change from baseline in LDL-C on day 510; time-adjusted percent change from baseline in LDL-C between day 90 and day 540
- Follow-up
- Day 510; days 90–540
Primary result
As reported in the abstract
Inclisiran
−39.7
percent change at day 510 · 95% CI −43.7–-35.7 · %
Placebo
8.2
percent change at day 510 · 95% CI 4.3–12.2 · %
Inclisiran vs PlaceboBetween-group difference, percentage points (95% CI)P value
day 510
−47.9(−53.5 to −42.3)
P < 0.001time-averaged, days 90–540
−44.3(−48.5 to −40.1)
P < 0.001Further findings
LDL-C by familial hypercholesterolemia genotype
- Robust LDL-C reductions in all genotypes
Safety
Adverse events and serious adverse events
- Similar in the two groups
Original publication
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia. N Engl J Med. Published 18 March 2020. doi:10.1056/nejmoa1913805
Summarised from the published abstract; every number is checked against it