Evidence map›Paper›PMID 40211340›Full record

Trial reportEuropean journal of medical research2025

Lomitapide modifies high-density lipoprotein function in homozygous familial hypercholesterolaemia.

Anouar Hafiane, Annalisa Ronca, Matteo Incerti, Alessandra Rossi, Matteo Manfredini, Elda Favari

Registry-linked trialAbstract readClinical Trial, Phase III
PubMed Full text at PMC Publisher
In one paragraph

Trial report in European journal of medical research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00730236 (A Phase III Study of Microsomal Triglyceride Transfer Protein), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

NCT00730236 phase3completednot on this map

A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy

TypeinterventionalSponsorAegerion Pharmaceuticals, Inc.Ran2007 to 2011Enrolled29ConditionsHomozygous Familial HypercholesterolemiaArmsAEGR-733
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Review
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Anouar HafianeDepartment of Medicine, Faculty of Medicine, Research Institute of the McGill University Health Centre, 1001 Boul Decarie, Montreal, Québec, H3A 1A1, Canada. anouar.hafiane@mail.mcgill.ca.
Annalisa RoncaDepartment of Food and Drug, University of Parma, Parco Area Delle Scienze, 27/A, 43124, Parma, Italy.
Matteo IncertiDepartment of Food and Drug, University of Parma, Parco Area Delle Scienze, 27/A, 43124, Parma, Italy.
Alessandra RossiDepartment of Food and Drug, University of Parma, Parco Area Delle Scienze, 27/A, 43124, Parma, Italy.
Matteo ManfrediniDepartment of Chemistry, Life Science, and Environmental Sustainability, University of Parma, Parma, Italy.
Elda FavariDepartment of Food and Drug, University of Parma, Parco Area Delle Scienze, 27/A, 43124, Parma, Italy. elda.favari@unipr.it.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundLomitapide reduces plasma low-density lipoprotein cholesterol (LDL-C) and is approved for the treatment of homozygous familial hypercholesterolemia (HoFH). This study aims to determine the effect of lomitapide on HDL and cholesterol efflux in a cohort of patients with HoFH. PATIENTS AND

methodsAnalysis included plasma samples from 17 HoFH patients enrolled in the lomitapide phase 3 Aegerion clinical study (NCT00730236). Samples taken at baseline (pre-lomitapide) and weeks 56 and 66 (assumed steady-state on lomitapide) were analyzed for HDL-C levels and cholesterol efflux capacity (CEC) pathways via ABCA1, ABCG1, and SR-BI cholesterol uptake.

resultsTreatment with lomitapide is associated with a statistically significant decrease of both LDL-C and apo B when compared to baseline levels, p < 0.01. However, the reduction of Lp(a) appears only at a higher dose when compared to baseline (- 27% against values around -  55% for LDL-C and apo B). HDL-C shows a small 4.2% increase between the baseline and the treatment with a high dosage of lomitapide, while apo A-I displays an opposite small 3% decrease. Total efflux and ABCA1 mediated CEC decreased especially at higher dosage of lomitapide, with marked dose-dependent increase of SR-BI cholesterol uptake (+ 21.4% and + 64.3%, respectively, at a low and high dosages of lomitapide). However, ABCG1 did not change consistently.

conclusionsOur report raises the hypothesis that lomitapide promotes lipidation of HDL particles independently of ABCA1 and ABCG1 through a process involving SR-BI pathway. This effect impairs the total efflux process suggesting that lomitapide drives the reverse cholesterol transport through SR-BI receptors in HoFH patients.

Indexed as

Anticholesteremic AgentsBenzimidazolesHyperlipoproteinemia Type IILipoproteins, HDLAdultATP Binding Cassette Transporter 1ATP Binding Cassette Transporter, Subfamily G, Member 1Cholesterol, HDLCholesterol, LDLFemaleHomozygoteHumansMaleMiddle AgedScavenger Receptors, Class BABCA1 protein, humanABCG1 protein, humanAnticholesteremic AgentsATP Binding Cassette Transporter 1ATP Binding Cassette Transporter, Subfamily G, Member 1BenzimidazolesBMS201038Cholesterol, HDLCholesterol, LDLLipoproteins, HDLScavenger Receptors, Class BApo BCholesterol efflux capacityHDL cholesterolHomozygous familial hypercholesterolaemiaLDL cholesterolLomitapide

Identifiers

PMID40211340
PMCPMC11987243

What Socratic holds

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LicenceCC BY-NC-ND
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.