drugset / Trial / NCT04659863
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
RandomizedParallel-groupDouble-blindTreatment
Summary
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).
Timeline
- Start
- 2021-02-16
- Primary completion
- 2023-10-30
- Completion
- 2024-11-18
Outcome
Met primary endpoint
registry analysis (other test); Part 1- Inclisiran vs Part 1 - Placebo; Mean Difference (Net) -33.25 (95% CI -59.17 to -7.34) NCT04659863 ↗
paper The placebo-adjusted mean (95% CI) percentage change in LDL-C from baseline to day 330 was -33.3% (-59.2% to -7.3%). PMID 40391436 ↗
Publications
- Wiegman A, Peterson AL, Hegele RA, Bruckert E, Schweizer A, Lesogor A, Wang Y, Defesche J. 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. 2025 Jun 24;151(25):1758-1766. doi: 10.1161/CIRCULATIONAHA.124.073233. Epub 2025 May 20.
- Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
- Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Inclisiran | Small interfering RNA (siRNA) | 300 mg | Subcutaneous |