Actively Recruiting
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Led by Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) · Updated on 2026-05-14
52
Participants Needed
13
Research Sites
144 weeks
Total Duration
On this page
Sponsors
F
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
Lead Sponsor
U
Ultragenyx Pharmaceutical Inc
Collaborating Sponsor
AI-Summary
What this Trial Is About
This observational, multicenter, retrospective and prospective study aims to evaluate the impact of intensified lipid-lowering therapy including Evinacumab on coronary atherosclerotic plaque burden in patients with Homozygous Familial Hypercholesterolemia (HoFH). HoFH is a rare genetic disorder characterized by extremely elevated low-density lipoprotein cholesterol (LDL-C) levels from early life and a markedly increased risk of premature atherosclerotic cardiovascular disease. Despite combination lipid-lowering therapy, many patients do not achieve recommended LDL-C targets and remain at high cardiovascular risk. Evinacumab, a monoclonal antibody targeting angiopoietin-like protein 3 (ANGPTL3), has demonstrated significant LDL-C reduction in clinical trials. However, real-world evidence on its impact on coronary plaque progression is limited. The study will compare HoFH patients receiving intensified lipid-lowering therapy including Evinacumab with patients receiving conventional lipid-lowering therapy without Evinacumab. Coronary plaque burden and phenotype will be assessed using coronary computed tomography angiography (CCTA) performed as part of routine clinical practice. Approximately 52 patients will be enrolled across European centers. The primary objective is to evaluate changes in non-calcified coronary plaque volume between baseline and 18-24 months' follow-up. Secondary objectives include evaluation of total plaque burden, high-risk plaque characteristics, and LDL-C reduction. Exploratory analyses will assess patient-reported outcomes, pericoronary adipose tissue characteristics, and supravalvular atherosclerosis. All data are collected from routine clinical care. No additional procedures are mandated by the protocol. This study aims to generate real-world imaging evidence on the effect of intensified lipid-lowering therapy including Evinacumab on coronary atherosclerosis in HoFH.
CONDITIONS
Official Title
Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willing and able to provide written informed consent or assent for use of retrospective and prospective data
- Male or female patients aged 12 years or older at enrollment (18 years or older in Italy and France)
- Clinical or genetic diagnosis of Homozygous Familial Hypercholesterolemia (HoFH)
- For intensified treatment group: Initiated Evinacumab as add-on therapy within 24 months before enrollment with stable background lipid-lowering treatment for at least 30 days prior
- For intensified treatment group: Availability of baseline CCTA performed at least 6 months before or 1 month after Evinacumab start and follow-up CCTA 18-24 months after
- For intensified treatment group: LDL cholesterol of 140 mg/dl or higher despite lipid-lowering treatment
- For conventional treatment group: On stable standard lipid-lowering therapy for at least 30 days before baseline CCTA and follow-up CCTA 18-24 months later
- For conventional treatment group: Required lipid-lowering therapies include statins, ezetimibe, and PCSK9 inhibitors unless discontinued due to insufficient LDL reduction
- For conventional treatment group: Optional stable lipoprotein apheresis or lomitapide for at least 3 months
- For conventional treatment group: LDL cholesterol of 140 mg/dl or higher despite lipid-lowering treatment
You will not qualify if you...
- Participation in a clinical trial with an investigational drug within the last 6 months
- Treatment outside of approved Evinacumab indication (for intensified group)
- Inability to access adequate retrospective clinical data
- Inability or unwillingness to provide informed consent or refusal to participate
- Previous multi-vessel coronary artery bypass grafting (single-vessel CABG allowed)
- Pregnancy at time of lipid-lowering therapy administration
- Moderate to severe kidney impairment, end-stage renal disease, kidney transplant, or ongoing chronic renal replacement therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Unité de Lipidologie et Prévention Cardiovasculaire, Centre de Compétence Dyslipidémies Rares (CEDRA), Service de Nutrition, Hôpital Pitié-Salpétriêre
Paris, France
Not Yet Recruiting
2
Dipartimento Scienze-Cardiovascolari, AO "Sant'Anna e San Sebastiano" di Caserta
Caserta, Italy
Actively Recruiting
3
U.O.C. di Medicina Interna, P.O. Nesima, ARNAS Garibaldi
Catania, Italy
Not Yet Recruiting
4
Nefrologia e Emodialisi, Centro Aterosclerosi e Dislipidemie, Ospedale Bassini, ASST Nord Milano
Cinisello Balsamo, Italy
Actively Recruiting
5
Malattie Aterotrombotiche, Azienda Ospedaliero Universitaria Careggi
Florence, Italy
Actively Recruiting
6
DAI di Medicina Clinica, Centro di Riferimento Regionale di Lipidologia e Dislipidemie, AOU Federico II di Napoli
Naples, Italy
Actively Recruiting
7
UOC Clinica Medica I, AOU di Padova
Padova, Italy
Not Yet Recruiting
8
U.O. Astanteria/MCAU AOU, Policlinico "Paolo Giaccone" di Palermo
Palermo, Italy
Actively Recruiting
9
Centro per le Malattie Rare del Metabolismo dei Lipidi, Unità di Medicina Interna e Malattie Metaboliche, Dipartimento di Medicina Traslazionale e di Precisione Sapienza, Università di Roma
Roma, Italy
Actively Recruiting
10
Medicina Interna, Ospedale Molinette, AOU Città della Salute e della Scienza
Torino, Italy
Not Yet Recruiting
11
Amsterdam University Medical Center, Amsterdam UMC, locatie AMC
Amsterdam, Netherlands
Actively Recruiting
12
Erasmus University Medical Center, Dr. Molewaterplein 40
Rotterdam, Netherlands
Not Yet Recruiting
13
Ege University,Director of Lipid and Prevention Clinic, Department of Cardiology
Bornova, Turkey (Türkiye)
Actively Recruiting
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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