Familial HypercholesterolemiaNCT03398954

a Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia

An observational study of Familial Hypercholesterolemia, run by China Cardiovascular Association at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Trial summary

1. Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot outpatient department of China. 2. Read more about this study

Who is it for?: Any age
Conditions the study is for: Familial Hypercholesterolemia
How many people: 1,128 participants
Where: 1 siteChina
Study dates: 18 Jan 2018to 30 Dec 2019
How long will I take part?: 2 Years
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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: For the primary objective the study population will comprise all patients with a documented LDL-C>4.65mmol/L if statin naïve and LDL-C>3.7mmol/L if on statin treatment before enrollment in the outpatient department of pilot hospitals from Sept. 2017-Sept. 2019.

Who may be able to join

Written inform consent provided.
Male and female cardiovascular outpatients and inpatients with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment before enrollment during Sept.2017 to Sept. 2019.

Who cannot join

Subjects who cannot understand study procedure
Subjects diagnosed as secondary dyslipidemia

Trial locations and contacts

1 site in 1 country.

huo yong, master contact

13901333060

drhuoyong@163.com

qi li tong, doctor contact

13671360078

qilitong2013@aliyun.com

China1 site, 1 recruiting

Beijing Anzhen Hospital
Beijing, Beijing Municipality
lvya wang
lvya wang
Recruiting

Other trials in China

About this study

Official title: Screening Out Rate and Clinical Management of Familial Hypercholesterolemia： a Prospective Pilot Study in China Outpatient Department

Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot outpatient department of China.
Study Design The study is a prospective observational research study of clinical diagnoses FH patients in outpatient department in pilot hospitals to evaluate the screening out rate and the clinical feature and management of FH patients including HoFH, with applying China recent issued FH screening protocol.
Eligibility 3.1.Inclusion Criteria Written inform consent provided. Male and female cardiovascular outpatients and inpatients with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment before enrollment during Sept.2017 to Sept. 2019. 3.2Exclusion Criteria Subjects who cannot understand study procedure Subjects diagnosed as secondary dyslipidemia
Primary Endpoint

The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China.
The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result.
The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration.

Read the detailed description

Objective 1.1.Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot outpatient department of China. 1.2.Secondary Objective To describe parameters related to clinical management of diagnosed FH, including patient demographics and characteristics, LDL-C and triglyceride concentrations, use of lipid modifying therapies, and outcomes over time. 1.3.Exploratory To describe how China FH screening protocol fits in the practice when compared with DLCN. To explore the multi-disciplinary practical pattern of FH patient management
Endpoint(s)/Outcome(s) Assessment 2.1.Endpoint(s) 2.1.1.Primary Endpoint The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China. The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result. The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration. 2.1.2.Secondary Endpoint LDL-C level during the follow up period The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration during the follow up period. Achievement of 2016 China guideline defined target LDL-C levels; <2.6mmol/L (high risk), <1.8mmol/L (very high risk) Incidence of cardiovascular event (CV death, all-cause mortality, non-fatal MI, non-fatal stroke). 2.1.3Exploratory Endpoint The difference of FH screening out rate between China criteria and DLCN. 2.2.Study Parameters 2.2.1.Baseline Data Collection Demography data:Age,Gender, Personal history:Premature CHD history of the subject (male: <55yrs, female: <65yrs),Premature PAD or cerebral vascular disease of the subject (male: <55yrs, female: <65yrs),Smoking, Medical history:PAD,DM,Hypertension,Heart Failure,Stroke,MI,CABG/PTCA/PCI,CKD Family history:Premature CHD within 2nd grade relatives (1st grade relatives, male: <55yrs, female: <60yrs; 2nd grade relatives, male: <50yrs, female: <55yrs),FH diagnosis within 2nd grade relatives Physical exam:Height,Weight,BMI,Corneal Arcus,Xanthoma,Achilles tender thickness Lab:LDL-C,HDL-C ,TC,TG,Lp(a),ALT,AST,CK,Glucose,Creatinine,Urea nitrogen,Inflammatory biomarker, including hs CRP ECG Cardiovascular imagine:CAG(optional),Myocardial radionuclide imaging (optional),UCG(annually),Carotid plaques measure(annually) Lipid modification therapy:Statin(type, dosage),Non-Statin(type, dosage) Apheresis 2.2.2Study Follow Up Period Data Collection Lab:LDL-C,HDL-C,TC,TG,Lp(a),ALT,AST,CK,Glucose,Creatinine,Urea nitrogen Inflammatory biomarker, including hs CRP Cardiovascular imagine:CAG(optional),Myocardial radionuclide imaging (optional),UCG(annually),Carotid plaques(annually) Lipid modification therapy:Statin(type, dosage),Non-Statin(type, dosage) Apheresis; Achievement of 2016 China guideline defined target LDL-C levels; <2.6mmol/L (high risk), <1.8mmol/L (very high risk); Incidence of cardiovascular event (CV death, all-cause mortality, non-fatal MI, non-fatal stroke).
STATISTICAL ANALYSIS 3.1.Statistical Inference No formal hypothesis will be tested in this study. This study will estimate the prevalence of Familial Hypercholesterolemia and the related 95% confidence interval in the China outpatient population in pilot hospitals. 3.2.Sample Size Considerations Based on initial feasibility analyses,revealed an approximate annual patient population in cardiology outpatients is 1,000pts/month(50pts/day*20days) with lipid profile measured to be screened. It is estimated that about 3000 patients out of cardiology department will measure their lipid profile in the clinical laboratory, the working days for the outpatient department is defined as 10mon, then total patients number in the outpatient will be about 4000pts/mon/center*10mon=40,000/center/yr. The prevalence of suspected patients based on LDL-C level is 0.47%, then 188 clinical diagnosed patients/center/year to be transited to the follow up period. Based on the planned site number of 6, total patient to be enrolled to this study will be 1128. Based on the prevalence of HoFH is near 1/160,000~1/300,000, it is estimated that 1~2 HoFH patients will be identified from this patient population. 3.3.Planned Analyses All summaries of the data will be descriptive in nature. For categorical variables (including the primary outcome measure), the frequency and percentage, with 95% confidence interval, will be given. Summary statistics for continuous variables will include the number of subjects, mean, median, standard deviation or standard error, 25th percentile (Q1), 75th percentile (Q3), minimum, and maximum. The outcomes over time data will be evaluated using time to event analyses/Cox proportional hazards models. All planned analyses will be descriptive. SAS version 9.2 (or a newer version) will be used for all Data Management and Statistical Analyses. 3.3.1.Description of Study Enrollment All subjects enrolled into the study who meet the inclusion criteria will be included in the full analysis set and will be included in all summaries and analyses. 3.3.2.Description of Subject/Patient Characteristics Clinical characteristics of subjects in the study will be summarized. Measurements such as age will be based on the status at the start of the observation period for the subject, and clinical characteristics such as CV events will be captured throughout the 3.3.3.Primary Analysis Primary Objective -To estimate the screen out rate of clinical diagnosed familial hypercholesterolemia in outpatient department of China. Prevalence: Prevalence of suspected FH will be estimated using the patients' data who visited the outpatients department in pilot hospitals and with measurable LDL-C data and validated by China criteria and DNCL respectively. Prevalence will be reported annually for the years 2017-2019. -To describe parameters related to clinical management of diagnosed FH, including patient demographics and characteristics, LDL-C and triglyceride concentrations, use of lipid modifying therapies, and outcomes over time. Patient demographics: Analyses of prevalence will be estimated for the following demographic characteristics: age, gender, and region. Patient clinical characteristics: The proportion of patients, calculated as percentage of patients, for each of the following: BMI (obese/not obese), diabetes, CKD, hypertension, any CVD history / number of CV events, smoking status (current / previous). These will be measured both at baseline (time of FH diagnosis). Current use of lipid-modifying therapies over time: Proportion of patients using LMT. Analyses by type, dose and frequency will analysis with description statistical method. Secondary Objective

LDL-C concentrations over time. The LDL-C level will be follow up to the end of study.
Use of lipid-modifying therapies over time。 Proportion of patients using LMT. Analyses by type, dose and frequency, and the proportion of patients on a high-intensity statin regime and with high LDL-C will be performed.
Proportion of patients within and at distance from 2016 China guideline's LDL-C target. This will be calculated as the percentage of patients within 2016 China guideline LDL-C target (<2.6 mmol/L, <1.8 mmol/L). LDL-C measurements will be followed up to the end of study. -Incidence of fatal and non-fatal cardiovascular events The proportion of patients having a fatal or non-fatal cardiovascular event will be calculated. Since there may be competing risks with fatal / non-fatal events, analyses will be performed separately, and in combination. All events occurring from date of enrollment of the study onwards will be included. 3.3.4.General Considerations The first primary objective and all secondary objectives will be addressed through descriptive statistics. The CV outcome will be evaluated using time to event analyses. 3.3.5.Missing or Incomplete Data and Lost to Follow-up There are two types of data in this study. For different data types, we will apply different methods to handle missing data. For Medical records abstraction: The missing age values will be imputed as the overall median to avoid case-wise deletions. For some of the patients, certain physiological indicators were not measured during hospitalization. These variables will be treated as a specific "unmeasured" subgroup with clinical significance because not measured is not equal to "missing" and may influence the outcomes. If we fit these variables as continuous in the models, we could only arbitrarily consider these "unmeasured" as missing value as inappropriate. Imputation also has limitations. Thus, to keep the models consistent with the features of data, we will transform certain continuous variables into categorical ones, created dummy variables accordingly, and including a specific category to indicate unmeasured. For Baseline and follow-up questionnaires: Site investigators completed the questionnaires in an electronic data capture program on a tablet computer (which allows real-time logic checks to ensure the accuracy and completeness of data). All questions were required to be answered, except those that were not applicable. There were options of "refuse to answer" and "don't know" for each question.

SAFETY DATA COLLECTION, RECORDING, AND REPORTING Due to the non-interventional nature of this study, no pro-active safety data collection will take place. Only spontaneously mentioned safety events and in-hospital clinical events will be collected in this study, and reported follow by local regulation.

Conditions: Familial Hypercholesterolemia
Medical subject headings: Hyperlipoproteinemia Type II

What is being measured

The primary outcome is the main question the study is designed to answer.

The screening out rate of FH
The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China.
Measured: 2 years
The clinical characteristics of clinical diagnosed FH patients
The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result
Measured: 2 years
The pharmaceutical therapy for clinical diagnosed FH patients
The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration
Measured: 2 years

Secondary outcomes (4)

LDL-C concentrations over time
LDL-C level during the follow up period
Measured: 2 years
Use of lipid-modifying therapies over time
The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration during the follow up period
Measured: 2 years
Proportion of patients within and at distance from 2016 China guideline's LDL-C target
Achievement of 2016 China guideline defined target LDL-C levels; <2.6mmol/L (high risk), <1.8mmol/L (very high risk)
Measured: 2 years
Incidence of cardiovascular event
Incidence of cardiovascular event (CV death, all-cause mortality, non-fatal MI, non-fatal stroke)
Measured: 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Jan 201830 Dec 2019

First posted16 Jan 2018
Study start18 Jan 2018
Primary completion30 Dec 2019 (estimated)
Study completion30 Dec 2019 (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 Familial HypercholesterolemiaStudies of the same condition, open and under way.
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: 1,128 participants (estimated)
Target duration: 2 Years

IDs, dates and publications

ClinicalTrials.gov ID: NCT03398954
Sponsor's protocol ID: 20177276
Status last verified: Jan 2018
First submitted: 20 Dec 2017
Last updated: 12 Mar 2018
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Qureshi N, Da Silva MLR, Abdul-Hamid H, Weng SF, Kai J, Leonardi-Bee J. Strategies for screening for familial hypercholesterolaemia in primary care and other community settings. Cochrane Database Syst Rev. 2021 Oct 7;10(10):CD012985. doi: 10.1002/14651858.CD012985.pub2.
PubMed 34617591

Want to know more?

huo yong, master

13901333060

drhuoyong@163.com

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
Hypercholesterolaemia, Familial Hypercholesterolaemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia

All Familial Hypercholesterolemia trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Screening Out Rate and Clinical Management of Familial Hypercholesterolemia： a Prospective Pilot Study in China Outpatient Department
ClinicalTrials.gov ID: NCT03398954
Acronym / sponsor ID: 20177276
Conditions: Familial Hypercholesterolemia
Study type: Observational
Recruitment status: Unknown status
Enrolment: 1,128 participants (estimated)
Lead sponsor: China Cardiovascular AssociationUniversity, hospital or other
Collaborators: Amgen
Study start: 18 Jan 2018 (actual)
Primary completion: 30 Dec 2019 (estimated)
Study completion: 30 Dec 2019 (estimated)
First posted: 16 Jan 2018 (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.

Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot outpatient department of China.
Study Design The study is a prospective observational research study of clinical diagnoses FH patients in outpatient department in pilot hospitals to evaluate the screening out rate and the clinical feature and management of FH patients including HoFH, with applying China recent issued FH screening protocol.
Eligibility 3.1.Inclusion Criteria Written inform consent provided. Male and female cardiovascular outpatients and inpatients with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment before enrollment during Sept.2017 to Sept. 2019. 3.2Exclusion Criteria Subjects who cannot understand study procedure Subjects diagnosed as secondary dyslipidemia
Primary Endpoint

The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China.
The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result.
The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration.

Detailed description

Objective 1.1.Primary Objective To estimate the prevalence of clinical diagnosed familial hypercholesterolemia, as well as the clinical characteristics and current treatment, with applying China recent issued FH screening protocol in pilot outpatient department of China. 1.2.Secondary Objective To describe parameters related to clinical management of diagnosed FH, including patient demographics and characteristics, LDL-C and triglyceride concentrations, use of lipid modifying therapies, and outcomes over time. 1.3.Exploratory To describe how China FH screening protocol fits in the practice when compared with DLCN. To explore the multi-disciplinary practical pattern of FH patient management
Endpoint(s)/Outcome(s) Assessment 2.1.Endpoint(s) 2.1.1.Primary Endpoint The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China. The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result. The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration. 2.1.2.Secondary Endpoint LDL-C level during the follow up period The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration during the follow up period. Achievement of 2016 China guideline defined target LDL-C levels; <2.6mmol/L (high risk), <1.8mmol/L (very high risk) Incidence of cardiovascular event (CV death, all-cause mortality, non-fatal MI, non-fatal stroke). 2.1.3Exploratory Endpoint The difference of FH screening out rate between China criteria and DLCN. 2.2.Study Parameters 2.2.1.Baseline Data Collection Demography data:Age,Gender, Personal history:Premature CHD history of the subject (male: <55yrs, female: <65yrs),Premature PAD or cerebral vascular disease of the subject (male: <55yrs, female: <65yrs),Smoking, Medical history:PAD,DM,Hypertension,Heart Failure,Stroke,MI,CABG/PTCA/PCI,CKD Family history:Premature CHD within 2nd grade relatives (1st grade relatives, male: <55yrs, female: <60yrs; 2nd grade relatives, male: <50yrs, female: <55yrs),FH diagnosis within 2nd grade relatives Physical exam:Height,Weight,BMI,Corneal Arcus,Xanthoma,Achilles tender thickness Lab:LDL-C,HDL-C ,TC,TG,Lp(a),ALT,AST,CK,Glucose,Creatinine,Urea nitrogen,Inflammatory biomarker, including hs CRP ECG Cardiovascular imagine:CAG(optional),Myocardial radionuclide imaging (optional),UCG(annually),Carotid plaques measure(annually) Lipid modification therapy:Statin(type, dosage),Non-Statin(type, dosage) Apheresis 2.2.2Study Follow Up Period Data Collection Lab:LDL-C,HDL-C,TC,TG,Lp(a),ALT,AST,CK,Glucose,Creatinine,Urea nitrogen Inflammatory biomarker, including hs CRP Cardiovascular imagine:CAG(optional),Myocardial radionuclide imaging (optional),UCG(annually),Carotid plaques(annually) Lipid modification therapy:Statin(type, dosage),Non-Statin(type, dosage) Apheresis; Achievement of 2016 China guideline defined target LDL-C levels; <2.6mmol/L (high risk), <1.8mmol/L (very high risk); Incidence of cardiovascular event (CV death, all-cause mortality, non-fatal MI, non-fatal stroke).
STATISTICAL ANALYSIS 3.1.Statistical Inference No formal hypothesis will be tested in this study. This study will estimate the prevalence of Familial Hypercholesterolemia and the related 95% confidence interval in the China outpatient population in pilot hospitals. 3.2.Sample Size Considerations Based on initial feasibility analyses,revealed an approximate annual patient population in cardiology outpatients is 1,000pts/month(50pts/day*20days) with lipid profile measured to be screened. It is estimated that about 3000 patients out of cardiology department will measure their lipid profile in the clinical laboratory, the working days for the outpatient department is defined as 10mon, then total patients number in the outpatient will be about 4000pts/mon/center*10mon=40,000/center/yr. The prevalence of suspected patients based on LDL-C level is 0.47%, then 188 clinical diagnosed patients/center/year to be transited to the follow up period. Based on the planned site number of 6, total patient to be enrolled to this study will be 1128. Based on the prevalence of HoFH is near 1/160,000~1/300,000, it is estimated that 1~2 HoFH patients will be identified from this patient population. 3.3.Planned Analyses All summaries of the data will be descriptive in nature. For categorical variables (including the primary outcome measure), the frequency and percentage, with 95% confidence interval, will be given. Summary statistics for continuous variables will include the number of subjects, mean, median, standard deviation or standard error, 25th percentile (Q1), 75th percentile (Q3), minimum, and maximum. The outcomes over time data will be evaluated using time to event analyses/Cox proportional hazards models. All planned analyses will be descriptive. SAS version 9.2 (or a newer version) will be used for all Data Management and Statistical Analyses. 3.3.1.Description of Study Enrollment All subjects enrolled into the study who meet the inclusion criteria will be included in the full analysis set and will be included in all summaries and analyses. 3.3.2.Description of Subject/Patient Characteristics Clinical characteristics of subjects in the study will be summarized. Measurements such as age will be based on the status at the start of the observation period for the subject, and clinical characteristics such as CV events will be captured throughout the 3.3.3.Primary Analysis Primary Objective -To estimate the screen out rate of clinical diagnosed familial hypercholesterolemia in outpatient department of China. Prevalence: Prevalence of suspected FH will be estimated using the patients' data who visited the outpatients department in pilot hospitals and with measurable LDL-C data and validated by China criteria and DNCL respectively. Prevalence will be reported annually for the years 2017-2019. -To describe parameters related to clinical management of diagnosed FH, including patient demographics and characteristics, LDL-C and triglyceride concentrations, use of lipid modifying therapies, and outcomes over time. Patient demographics: Analyses of prevalence will be estimated for the following demographic characteristics: age, gender, and region. Patient clinical characteristics: The proportion of patients, calculated as percentage of patients, for each of the following: BMI (obese/not obese), diabetes, CKD, hypertension, any CVD history / number of CV events, smoking status (current / previous). These will be measured both at baseline (time of FH diagnosis). Current use of lipid-modifying therapies over time: Proportion of patients using LMT. Analyses by type, dose and frequency will analysis with description statistical method. Secondary Objective

LDL-C concentrations over time. The LDL-C level will be follow up to the end of study.
Use of lipid-modifying therapies over time。 Proportion of patients using LMT. Analyses by type, dose and frequency, and the proportion of patients on a high-intensity statin regime and with high LDL-C will be performed.
Proportion of patients within and at distance from 2016 China guideline's LDL-C target. This will be calculated as the percentage of patients within 2016 China guideline LDL-C target (<2.6 mmol/L, <1.8 mmol/L). LDL-C measurements will be followed up to the end of study. -Incidence of fatal and non-fatal cardiovascular events The proportion of patients having a fatal or non-fatal cardiovascular event will be calculated. Since there may be competing risks with fatal / non-fatal events, analyses will be performed separately, and in combination. All events occurring from date of enrollment of the study onwards will be included. 3.3.4.General Considerations The first primary objective and all secondary objectives will be addressed through descriptive statistics. The CV outcome will be evaluated using time to event analyses. 3.3.5.Missing or Incomplete Data and Lost to Follow-up There are two types of data in this study. For different data types, we will apply different methods to handle missing data. For Medical records abstraction: The missing age values will be imputed as the overall median to avoid case-wise deletions. For some of the patients, certain physiological indicators were not measured during hospitalization. These variables will be treated as a specific "unmeasured" subgroup with clinical significance because not measured is not equal to "missing" and may influence the outcomes. If we fit these variables as continuous in the models, we could only arbitrarily consider these "unmeasured" as missing value as inappropriate. Imputation also has limitations. Thus, to keep the models consistent with the features of data, we will transform certain continuous variables into categorical ones, created dummy variables accordingly, and including a specific category to indicate unmeasured. For Baseline and follow-up questionnaires: Site investigators completed the questionnaires in an electronic data capture program on a tablet computer (which allows real-time logic checks to ensure the accuracy and completeness of data). All questions were required to be answered, except those that were not applicable. There were options of "refuse to answer" and "don't know" for each question.

SAFETY DATA COLLECTION, RECORDING, AND REPORTING Due to the non-interventional nature of this study, no pro-active safety data collection will take place. Only spontaneously mentioned safety events and in-hospital clinical events will be collected in this study, and reported follow by local regulation.

Study design

Observational model: Case only
Time perspective: Prospective
Target follow-up: 2 Years

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: For the primary objective the study population will comprise all patients with a documented LDL-C>4.65mmol/L if statin naïve and LDL-C>3.7mmol/L if on statin treatment before enrollment in the outpatient department of pilot hospitals from Sept. 2017-Sept. 2019.

Key inclusion criteria (2 listed)

Written inform consent provided.
Male and female cardiovascular outpatients and inpatients with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment before enrollment during Sept.2017 to Sept. 2019.

Key exclusion criteria (2 listed)

Subjects who cannot understand study procedure
Subjects diagnosed as secondary dyslipidemia

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

The screening out rate of FH
Time frame: 2 years
The screening out rate of clinical diagnosed familial hypercholesterolemia, with applying China recent issued FH screening protocol in subjects with LDL-C>4.65mmol/L if statin naïve or LDL-C>3.7mmol/L if on statin treatment in pilot outpatient department of China.
The clinical characteristics of clinical diagnosed FH patients
Time frame: 2 years
The clinical characteristics of clinical diagnosed FH patients(including HoFH and HeFH), including: demography, medical history, family history, sign and symptoms, lab testing and cardiovascular imagine result
The pharmaceutical therapy for clinical diagnosed FH patients
Time frame: 2 years
The pharmaceutical therapy for clinical diagnosed FH patients (including HoFH and HeFH), including the type of medication, proportion for each medication, dosage and treatment duration

4 secondary outcomes

LDL-C concentrations over time
Time frame: 2 years
Use of lipid-modifying therapies over time
Time frame: 2 years
Proportion of patients within and at distance from 2016 China guideline's LDL-C target
Time frame: 2 years
Incidence of cardiovascular event
Time frame: 2 years

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2018.
Derived from this study: PubMed 34617591Qureshi N, Da Silva MLR, Abdul-Hamid H, Weng SF, Kai J, Leonardi-Bee J. Strategies for screening for familial hypercholesterolaemia in primary care and other community settings. Cochrane Database Syst Rev. 2021 Oct 7;10(10):CD012985. doi: 10.1002/14651858.CD012985.pub2.
Individual participant dataSharing planned: No

Related trials for Familial Hypercholesterolemia

Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
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Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
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Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia

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.

huo yong, master contact

13901333060

drhuoyong@163.com

qi li tong, doctor contact

13671360078

qilitong2013@aliyun.com

huo yong, master principal investigator

Peking University First Hospital

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 Familial Hypercholesterolemia, run by China Cardiovascular Association at 1 site in China.

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 2 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 12 Mar 2018. 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 China. 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?

Contact huo yong, master (13901333060, drhuoyong@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The screening out rate of FH (time frame: 2 years). The study lists 3 primary outcomes in all. 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 NCT03398954, 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 12 Mar 2018.