Design and rationale of the VICTORION-Difference study: A phase 4 randomized, double-blind, placebo-controlled clinical trial to assess inclisiran's early efficacy, safety, tolerability, as well as its impact on quality of life in individuals with hypercholesterolemia

Am Heart J. 2025 May 26:289:117-126. doi: 10.1016/j.ahj.2025.05.014. Online ahead of print.

Abstract

Introduction: Twice-yearly inclisiran (after the initial and 3-month doses) provides effective and sustained low-density lipoprotein cholesterol (LDL-C) reduction in individuals with hyperlipidemia with a favorable long-term safety profile. Limited studies have assessed the impact of inclisiran on the quality of life (QoL) of patients with atherosclerotic cardiovascular disease (ASCVD) or ASCVD-risk equivalent. The VICTORION-Difference study will evaluate the early efficacy, safety, and QoL outcomes with inclisiran compared to placebo on top of individually optimized lipid-lowering therapy.

Methods and analysis: This phase 4, randomized, double-blind, placebo-controlled trial includes participants aged ≥18 years with hypercholesterolemia at high or very high cardiovascular (CV) risk despite being treated with an individualized maximally tolerated statin dose. Participants are randomized 1:1 to receive either subcutaneous injections of 300-mg inclisiran sodium (equivalent to 284-mg inclisiran) or placebo. In addition to the treatment with inclisiran/placebo, open-label rosuvastatin (starting dose of 5 mg/day or 10 mg/day) is sequentially and optimally titrated to the maximally tolerated dose, if the individual LDL-C goals are not achieved. The primary objective is to assess the achievement of LDL-C goals at day 90. Secondary objectives include the assessment of muscle-related adverse events, pain-related QoL scores, and mean LDL-C reduction.

Ethics and dissemination: The results of the study will be disseminated through peer-reviewed journals, conference presentations, and other scientific forums to inform clinical practice and future research in the field of hypercholesterolemia and CV disease management.

Trial registration number: Unique identifier: NCT05192941.

Associated data

  • ClinicalTrials.gov/NCT05192941