Trial reportAdvances in therapy2025
Evinacumab for Homozygous Familial Hypercholesterolemia: The Italian Cohort of the ELIPSE HoFH Study.
Trial report in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03399786 (A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia), which is not on this map. Cited by 2 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Who cites it
2 citing papers in PubMed.
- Review
- Homozygous familial hypercholesterolemia, experience with Evinacumab treatment in two Mexican pediatric patients: case report.Frontiers in genetics · 2026Article
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionHomozygous familial hypercholesterolemia (HoFH) is a severe rare genetic disorder characterized by elevated plasma low-density lipoprotein (LDL) cholesterol levels. Here, we report data from the Italian cohort of the Evinacumab Lipid Studies in Patients with Homozygous Familial Hypercholesterolemia (ELIPSE HoFH) trial.
methodsELIPSE HoFH was conducted at 30 sites in 11 countries, with 2-10 patients enrolled per country. The study included patients aged ≥ 12 years with LDL cholesterol ≥ 70 mg/dl (1.8 mmol per liter) at screening despite stable maximally tolerated lipid-lowering therapy. Patients were randomly assigned evinacumab (15 mg/kg every 4 weeks) or matching placebo for 24 weeks, with an option for a 24-week open-label extension or follow-up period thereafter. The Italian cohort included seven patients assigned to evinacumab.
resultsFive patients (3 males and 2 females) received evinacumab and were included in this report. Substantial and consistent reductions in LDL cholesterol from baseline levels were observed in all patients at all follow-up time points. Overall, an 84.5% decrease in median (range) LDL cholesterol was observed, from 323 (203-587) mg/dl in 2016 to 50.0 (13-103) mg/dl (P = 0.043) in 2019, with LDL cholesterol levels stable through 2023. Total cholesterol, high-density lipoprotein (HDL) cholesterol, non-HDL cholesterol, and triglycerides decreased markedly over time. Evinacumab was well tolerated, with no treatment-related adverse events reported.
conclusionEvinacumab substantially lowered LDL cholesterol levels in patients with HoFH regardless of the degree of LDL receptor function, with low levels sustained over 5 years of follow-up.
trial registrationClinicalTrials.gov identifier NCT03399786 registered 16 January 2018.
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