Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia

NCT ID: NCT01878604

Last Updated: 2017-02-20

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

5 participants

Study Classification

OBSERVATIONAL

Study Start Date

2001-10-31

Study Completion Date

2015-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Identify new or novel genes which may impact on cholesterol level, and establish the relationship between those gene mutations with atherosclerosis, as well as responses to lipid-lowering drugs.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

To better understand the genetics basis for LDL-C elevation and develop an optimized lipid-lowering strategy, we propose to do the following studies:

1. To establish a China HoFH registry, and collect DNA and blood samples from all available family members of each proband (pedigrees);
2. To detect gene mutations known to cause FH and identify family suitable for future whole genome sequencing aimed to identify novel genes controlling cholesterol levels.

3.To establish the relationship between types of gene mutations and lipid and atherosclerosis profile, as well as responses to lipid-lowering agents.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Homozygous Familial Hypercholesterolemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Homozygous Familial Hypercholesterolemia

Gene Analysis for Homozygous Familial Hypercholesterolemia cases

Gene analysis

Intervention Type GENETIC

Gene analysis

Historical data of lipid-lowering drug administration

Intervention Type OTHER

Collecting historical data of lipid-lowering drug administration

Historical data of plasma lipids, xanthoma changes

Intervention Type OTHER

Collecting historical data of plasma lipids and xanthoma changes

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Gene analysis

Gene analysis

Intervention Type GENETIC

Historical data of lipid-lowering drug administration

Collecting historical data of lipid-lowering drug administration

Intervention Type OTHER

Historical data of plasma lipids, xanthoma changes

Collecting historical data of plasma lipids and xanthoma changes

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Patients of any age and sex who meet clinical or genetic criteria for hoFH as follows:

* Cutaneous xanthomata before the age of ten years
* LDLC \> 13 mmol/L before treatment or \> 7.76 mmol/L despite treatment
* Phenotypic features in keeping with HeFH in both parents

Exclusion Criteria

Inability of patient, or, if less than 18, a parent, to sign informed consent.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Central South University

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Shuiping Zhao

Chief of Cardiology Department, 2nd Xiangya Hospital

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Shuiping Zhao, Doctor

Role: PRINCIPAL_INVESTIGATOR

Central South University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Cardiology department of 2nd Xiangya Hospital

Changsha, Hunan, China

Site Status

Countries

Review the countries where the study has at least one active or historical site.

China

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

MISP50469

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Genetic Study of Sitosterolemia
NCT00004481 COMPLETED