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): A Real World, Prospective and Retrospective, Observational Study
Led by Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) · Updated on 2026-05-14
52
Participants Needed
13
Research Sites
74 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
Researchers are evaluating the impact of intensified lipid-lowering therapy including Evinacumab on coronary atherosclerotic plaque burden in patients with Homozygous Familial Hypercholesterolemia (HoFH), a rare genetic disorder marked by very high LDL cholesterol from early life and increased risk of early heart disease. This observational study includes both retrospective and prospective data, aiming to understand how Evinacumab affects plaque progression compared to conventional treatments. Participants are divided into two groups based on routine clinical care: one receiving intensified therapy with Evinacumab added to stable lipid-lowering treatments, and the other receiving conventional lipid-lowering therapy without Evinacumab. Coronary computed tomography angiography (CCTA) scans, performed as part of routine care, will assess plaque volume and characteristics over 18-24 months. The study observes changes in non-calcified plaque volume, total plaque burden, high-risk plaque features, and LDL cholesterol levels. Participants will provide data from past and future routine clinical care, including CCTA imaging and laboratory tests. Patient-reported outcomes and adipose tissue characteristics around coronary arteries will also be evaluated. Researchers will track changes over time, focusing on plaque stability or regression, with safety information collected from medical records. The study is expected to last up to 24 months of follow-up with about 52 patients enrolled across multiple European centers.
CONDITIONS
Brief 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 data use
- 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 within 24 months before enrollment as add-on to stable lipid-lowering therapy
- For conventional treatment group: on stable standard lipid-lowering therapy for at least 30 days before baseline coronary CT scan
- Availability of baseline and follow-up coronary CT angiography (CCTA) scans performed at routine care intervals
- LDL cholesterol level of 140 mg/dl or higher despite lipid-lowering treatment
You will not qualify if you...
- Participation in a clinical trial with investigational drugs within the last 6 months
- Treatment outside approved Evinacumab indication (intensified group only)
- 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 the time of lipid-lowering treatment administration
- Moderate to severe kidney impairment, end-stage renal disease, kidney transplant, or ongoing dialysis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months prospective follow-up plus up to 30 months retrospective data
Participants who undergo routine lipid-lowering therapy, including those initiating Evinacumab, are observed through their regular clinical care. Coronary CT angiography (CCTA) scans are performed at routine care intervals to assess coronary plaque volumes and characteristics.
CCTA scans and assessments at Baseline, 6 months, 12 months, and 18 to 24 months
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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