Hypercholesterolemia, FamilialNCT04313270
Subclinical Atherosclerosis in Patients With Familial Hypercholesterolemia Treated With Evolocumab®
An observational study of Hypercholesterolemia, Familial, run by Federico II University at 1 site in Italy.
Trial summary
Protein convertase subtilisin kexin type 9 (PCSK-9) inhibitors demonstrated efficacy in cholesterol reduction and in the prevention of cardiovascular events. The investigators will evaluate changes in lipid profile, oxidation markers and subclinical atherosclerosis in patients with familial hypercholesterolemia (FH)… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypercholesterolemia, Familial
- What is being tested?
- Evolocumab
- How many people
- 25 participants
- Where
- 1 siteItaly
- Study dates
- 1 Dec 2017to 1 Dec 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: consecutive patients attending the lipid clinic of the Department of Clinical Medicine and Surgery, Federico II University Hospital with very high levels of LDL-C (above the 95th percentile when compared with a sex- and age-matched general population), normal triglyceride levels and presumed autosomal dominant transmission of hypercholesterolemia in the family were screened for inclusion in the present study
Who may be able to join
- diagnosis of FH (clinical and/or genetic)
- eligibility of patients to start a treatment with PCSK-9 according to 2016 ESC guidelines.
Who cannot join
- age < 18 years
- inability to understand or sign the informed consent
- high level of transaminases ( >3x upper normal limit)
- hypertriglyceridemia ( >150 mg/dl)
- end-stage renal disease (filtration rate < 30 ml/min/mq)
- current malignant disease or a diagnosis of malignancy in the 2 years prior to the first visit
- previous exposure to PCSK-9 inhibitors
- presence of hypercholesterolemia secondary to other causes (hypothyroidism, hormone therapies, corticosteroids etc.)
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Matteo Di Minno
Naples, 80131
Matteo Di Minno, MD, +390817464323, dario.diminno@hotmail.it
About this study
Protein convertase subtilisin kexin type 9 (PCSK-9) inhibitors demonstrated efficacy in cholesterol reduction and in the prevention of cardiovascular events. The investigators will evaluate changes in lipid profile, oxidation markers and subclinical atherosclerosis in patients with familial hypercholesterolemia (FH) during 12 weeks of treatment with a PCSK-9 inhibitor, Evolocumab®.
Read the detailed description
Several studies emphasize the role of high levels of low-density lipoprotein cholesterol (LDL-C) as the main causative factor in atherosclerosis development. Among patients with hypercholesterolemia, those with very high levels of LDL-C exhibit increased prevalence of subclinical atherosclerosis and a higher atherosclerosis progression, thus leading to a significantly higher CV risk. Endothelial dysfunction is the earliest stage of the atherosclerotic process and even a trigger of CV events. Flow-mediated dilation (FMD) is widely accepted as an accurate and non-invasive method to assess vascular reactivity and, in turn, as a surrogate marker of subclinical atherosclerosis and an independent predictor of CV events. It's well known that hypercholesterolemia has been associated with decreased endothelial function and increased oxidative stress. Although statin treatment represented for years the gold standard as lipid lowering therapy, the target LDL-C is not always achieved, mainly among patients with very high levels of LDL-C. More recently, PCSK-9 inhibitors demonstrated efficacy in LDL-C reduction, in the prevention from CV events and in atherosclerotic burden regression. Some data showed an effect of PCSK-9 inhibitors on endothelial function, but no evidence is available on effect on LDL subfractions. Small dense LDL (sd-LDL) are considered an emerging risk factor for cardiovascular disease due to a greater atherogenic potential [ ] and are important markers for predicting CV risk.
- Conditions
- Hypercholesterolemia, Familial
- Being tested
- Evolocumab
- Medical subject headings
- Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EvolocumabDrug
12 weeks of treatment with Evolocumab
| Study group | Role | What they receive |
|---|---|---|
| Patients with FH starting a treatment with Evolocumab® | Not described Receives: Drug: Evolocumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Subclinical atherosclerosis
evaluation of subclinical atherosclerosis in patients receiving Evolocumab
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2020
- Study start1 Dec 2017
- Primary completion1 Dec 2022 (estimated)
- Study completion1 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hypercholesterolemia, FamilialStudies of the same condition, open and under way.
- More trials run by Federico II UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Federico II University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Matteo Di Minno, Federico II University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04313270
- Sponsor's protocol ID
- 2015/261
- Status last verified
- Mar 2021
- First submitted
- 15 Mar 2020
- Last updated
- 8 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Iannuzzo G, Buonaiuto A, Calcaterra I, Gentile M, Forte F, Tripaldella M, Di Taranto MD, Giacobbe C, Fortunato G, Rubba PO, Di Minno MND. Association between causative mutations and response to PCSK9 inhibitor therapy in subjects with familial hypercholesterolemia: A single center real-world study. Nutr Metab Cardiovasc Dis. 2022 Mar;32(3):684-691. doi: 10.1016/j.numecd.2021.10.025. Epub 2021 Nov 11.
PubMed 34991937
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Subclinical Atherosclerosis in Patients With Familial Hypercholesterolemia Treated With Evolocumab®
- ClinicalTrials.gov ID
- NCT04313270
- Acronym / sponsor ID
- 2015/261
- Conditions
- Hypercholesterolemia, Familial
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Federico II UniversityUniversity, hospital or other
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 1 Dec 2022 (estimated)
- Study completion
- 1 Dec 2025 (estimated)
- First posted
- 18 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Protein convertase subtilisin kexin type 9 (PCSK-9) inhibitors demonstrated efficacy in cholesterol reduction and in the prevention of cardiovascular events. The investigators will evaluate changes in lipid profile, oxidation markers and subclinical atherosclerosis in patients with familial hypercholesterolemia (FH) during 12 weeks of treatment with a PCSK-9 inhibitor, Evolocumab®.
Detailed description
Several studies emphasize the role of high levels of low-density lipoprotein cholesterol (LDL-C) as the main causative factor in atherosclerosis development. Among patients with hypercholesterolemia, those with very high levels of LDL-C exhibit increased prevalence of subclinical atherosclerosis and a higher atherosclerosis progression, thus leading to a significantly higher CV risk. Endothelial dysfunction is the earliest stage of the atherosclerotic process and even a trigger of CV events. Flow-mediated dilation (FMD) is widely accepted as an accurate and non-invasive method to assess vascular reactivity and, in turn, as a surrogate marker of subclinical atherosclerosis and an independent predictor of CV events. It's well known that hypercholesterolemia has been associated with decreased endothelial function and increased oxidative stress. Although statin treatment represented for years the gold standard as lipid lowering therapy, the target LDL-C is not always achieved, mainly among patients with very high levels of LDL-C. More recently, PCSK-9 inhibitors demonstrated efficacy in LDL-C reduction, in the prevention from CV events and in atherosclerotic burden regression. Some data showed an effect of PCSK-9 inhibitors on endothelial function, but no evidence is available on effect on LDL subfractions. Small dense LDL (sd-LDL) are considered an emerging risk factor for cardiovascular disease due to a greater atherogenic potential [ ] and are important markers for predicting CV risk.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with FH starting a treatment with Evolocumab® | – | Drug: Evolocumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- consecutive patients attending the lipid clinic of the Department of Clinical Medicine and Surgery, Federico II University Hospital with very high levels of LDL-C (above the 95th percentile when compared with a sex- and age-matched general population), normal triglyceride levels and presumed autosomal dominant transmission of hypercholesterolemia in the family were screened for inclusion in the present study
Key inclusion criteria (2 listed)
- diagnosis of FH (clinical and/or genetic)
- eligibility of patients to start a treatment with PCSK-9 according to 2016 ESC guidelines.
Key exclusion criteria (8 listed)
- age < 18 years
- inability to understand or sign the informed consent
- high level of transaminases ( >3x upper normal limit)
- hypertriglyceridemia ( >150 mg/dl)
- end-stage renal disease (filtration rate < 30 ml/min/mq)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Evolocumab12 weeks of treatment with Evolocumab | Drug | Patients with FH starting a treatment with Evolocumab® |
Primary outcome
Subclinical atherosclerosis
Time frame: 12 weeks
evaluation of subclinical atherosclerosis in patients receiving Evolocumab
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Mar 2021.
- Derived from this study: PubMed 34991937Iannuzzo G, Buonaiuto A, Calcaterra I, Gentile M, Forte F, Tripaldella M, Di Taranto MD, Giacobbe C, Fortunato G, Rubba PO, Di Minno MND. Association between causative mutations and response to PCSK9 inhibitor therapy in subjects with familial hypercholesterolemia: A single center real-world study. Nutr Metab Cardiovasc Dis. 2022 Mar;32(3):684-691. doi: 10.1016/j.numecd.2021.10.025. Epub 2021 Nov 11.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypercholesterolemia, Familial, run by Federico II University at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Subclinical atherosclerosis (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04313270, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Mar 2021.