Prevention & lipidsRandomised controlled trial
Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial
- Randomised controlled trial
- double-blind
- placebo-controlled
- phase 3
- multicentre
- 2:1 randomisation
Authors' conclusion
In a 1-year randomized controlled study (part 1 of ORION-13), inclisiran was effective in lowering LDL-C in adolescents with HoFH and was well tolerated.
Study context
- Population
- Adolescents 12 to <18 years with genetically confirmed homozygous familial hypercholesterolemia (HoFH) (excluding LDLR null/null) and LDL-C >130 mg/dL on maximally tolerated statin
- Participants
- 13 adolescents
- Comparison
- Inclisiran vs placebo
- Inclisirann = 9
- Placebon = 4reference
- Primary endpoint
- Mean percentage change in LDL-C from baseline
- Follow-up
- Day 330
Primary result
Percentage change in LDL-C
Placebo-adjusted mean percentage change
−33.3%
95% CI −59.2% to −7.3%
Inclisiran vs Placebo
placebo-adjusted mean percentage change −33.3% (95% CI −59.2% to −7.3%)
As reported in the abstract
Further findings
>15% reduction in LDL-C
| Outcome | placebo-adjusted mean percentage change (95% CI) | |
|---|---|---|
| Percentage change in PCSK9 · day 330 | −60.2% (−79.8% to −40.7%) | |
| −100−50050Difference (linear scale) |
| Outcome | Inclisiran | Placebo | Estimate |
|---|---|---|---|
| >20% reduction in LDL-C | 55.6% | 0 events |
Apolipoprotein B, non-HDL cholesterol, total cholesterol · day 330
- Apolipoprotein B, placebo-adjusted change
- −23.0%
- Non-HDL cholesterol, placebo-adjusted change
- −32.7%
- Total cholesterol, placebo-adjusted change
- −27.8%
Safety
Serious adverse events, discontinuations due to adverse events, deaths
- No serious adverse events, discontinuations due to adverse events, or deaths
- No new safety findings
Original publication
Efficacy and Safety of Inclisiran in Adolescents With Genetically Confirmed Homozygous Familial Hypercholesterolemia: Results From the Double-Blind, Placebo-Controlled Part of the ORION-13 Randomized Trial. Circulation. Published 20 May 2025. doi:10.1161/circulationaha.124.073233
Summarised from the published abstract; every number is checked against it