Prevention & lipidsRandomised controlled trial
Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial
- Randomised controlled trial
- phase 3
- 2-part
- multicenter
- double-blind (part 1, 6 months)
- randomized 2:1
- open-label part 2 (18 months)
Authors' conclusion
Inclisiran treatment did not reduce LDL-C levels in patients with homozygous familial hypercholesterolemia despite substantial lowering of PCSK9 levels.
Study context
- Population
- Homozygous familial hypercholesterolemia with elevated LDL-C despite maximum tolerated LDL-C-lowering therapies with or without lipoprotein apheresis
- Participants
- 56 participants
- Comparison
- Inclisiran vs placebo
- Primary endpoint
- Percentage change in LDL-C from baseline
- Follow-up
- Day 150
Primary result
Placebo-corrected % change in LDL-C
Placebo-corrected percentage change
−1.68 %
95% CI −29.19% to 25.83%
P = 0.90
Inclisiran vs Placebo
placebo-corrected percentage change −1.68 % (95% CI −29.19% to 25.83%); P = 0.90
As reported in the abstract
Further findings
| Outcome | placebo-corrected percentage change (%) | P value |
|---|---|---|
| Placebo-corrected % change in PCSK9 · day 150 · sustained throughout the study | −60.6 | P < 0.0001 |
Other lipids and lipoproteins (apolipoprotein B, total cholesterol, non-HDL cholesterol)
- No statistically significant differences between inclisiran and placebo
Safety
Adverse events and serious adverse events · throughout the study
- Did not differ between inclisiran and placebo
Original publication
Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial. Circulation. Published 18 October 2023. doi:10.1161/circulationaha.122.063460
Summarised from the published abstract; every number is checked against it