China Registry for Genetic / Metabolic Liver Diseases

April 24, 2017 updated by: Jidong Jia, Beijing Friendship Hospital

A Nation-wide Hospital-based Registry:China Registry for Genetic / Metabolic Liver Diseases

CR-GMLD registry started on June 13, 2015 to collect cases of genetic/metabolic liver diseases from tertiary or secondary hospitals in mainland China. Demographics, diagnosis, laboratory test results, family history and prescriptions were recorded. Patients' whole blood and serum were collected for genetic testing and future researches. These patients will be followed-up every six to twelve months.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This web-based database was launched on June 13, 2015 and consists of tertiary or secondary hospitals with special interest and expertise on managing genetic/metabolic liver diseases patients across mainland China. The main inclusion criteria for this registration are patients who were diagnosed or possibly diagnosed with Wilson's disease, hereditary hemochromatosis, hereditary hyperbilirubinemias, inherited cholestatic liver disease or other genetic/metabolic liver diseases. At the first time of data entry, demographics, medical history, biochemistry and hematology results, radiology reports, diagnosis and treatment information were recorded. Patients' whole blood and serum were collected for molecular genetic testing and future researches. Then the registered patients will receive standard of care and be followed-up every 6 to 12 months. On each visit, biochemical, radiological reports, as well as clinical progress were recorded.

Study Type

Observational

Enrollment (Anticipated)

20000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100034
      • Beijing, Beijing, China, 100015
      • Beijing, Beijing, China, 100029
        • Recruiting
        • Beijing Anzhen Hospital, Capital Medical University
        • Contact:
      • Beijing, Beijing, China, 100069
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Recruiting
        • Nanfang Hospital of Southern Medical University
        • Contact:
    • Hebei
      • Shijiazhuang, Hebei, China, 050051
        • Recruiting
        • Hebei Medical University Third Hospital
        • Contact:
    • Henan
      • Zhengzhou, Henan, China, 450003
    • Shanghai
      • Shanghai, Shanghai, China, 200032
      • Shanghai, Shanghai, China, 201508
        • Recruiting
        • Jinshan Hospital of Fudan University
        • Contact:
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Women's and Children's Hospital, Sichuan University
        • Contact:
    • Tianjin
      • Tianjin, Tianjin, China, 300163
        • Recruiting
        • Affiliated Hospital, Logistics University of People's Armed Police Force
        • Contact:
    • Xinjiang
      • Urumqi, Xinjiang, China, 830099
        • Recruiting
        • Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The registry is a multicenter and observational study enrolling patients with genetic/metabolic liver disease across mailand China.

Description

Inclusion Criteria:

Patients who were diagnosed or possibly diagnosed with Wilson's disease, hereditary hemochromatosis, hereditary hyperbilirubinemias, inherited cholestatic liver disease or other genetic/metabolic liver diseases.

Exclusion Criteria:

Patients who are unable or unwilling to provide informed consent.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Wilson's Disease
Patients who were diagnosed or possibly diagnosed with Wilson's disease. The diagnosis can be made or possibly made on the basis of Wilson's disease scoring system proposed by the Working Party at the 8th International Meeting on Wilson's disease, Leipzig 2001.
Drug: Standard of care
Standard of care according to the updated national and/or international guidelines
Hereditary Hemochromatosis
Hereditary hemochromatosis can be clinically diagnosed if: ① transferrin saturation≥45% and/or elevated ferritin; ② iron overload in liver and/or spleen on magnetic resonance imaging (MRI) of liver or on liver histology; ③ exclude causes of secondary iron overload, such as alcoholic or other chronic liver disease, iron-overloading anemia, and parenteral iron overload.
Drug: Standard of care
Standard of care according to the updated national and/or international guidelines
Hereditary Hyperbilirubinemias
Hereditary hyperbilirubinemias involve four syndromes: Gilbert, Crigler-Najjar, Dubin-Johnson and Rotor, among which the first two are characterized by unconjugated hyperbilirubinemia and the second two by conjugated hyperbilirubinemia. Diagnosis of hereditary hyperbilirubinemia should exclude other causes of hyperbilirubinemia, such as obstructive bile duct (slerosing cholangitis, calculi, parasites), intrahepatic cholestasis(drugs, hepatitis, immune-mediated, infectious), acute or chronic hepatocellular injury(sepsis, parenteral nutrition, severe blood loss/hypotension, trauma, conjestive heart failure), increased bilirubin production(hemolysis, hematological disease), decreased bilirubin uptake (drugs, portosystemic shunting ), reduced conjugation activity (neonatal, thyroid disease, chronic hepatitis/inflammation, wilson's disease).
Drug: Standard of care
Standard of care according to the updated national and/or international guidelines
Inherited Cholestatic Liver Disease
Patients who were diagnosed or possibly diagnosed with Inherited cholestatic liver disease, including progressive familial intrahepatic cholestasis(PFIC) and benign recurrent intrahepatic cholestasis(BRIC).
Drug: Standard of care
Standard of care according to the updated national and/or international guidelines
Other genetic/metabolic liver diseases
Patients who were diagnosed or possibly diagnosed with genetic/metabolic liver diseases except for Wilson's disease, hereditary hemochromatosis, hereditary hyperbilirubinemias or inherited cholestatic liver disease.
Drug: Standard of care
Standard of care according to the updated national and/or international guidelines

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
rate of liver-related events of each disease.
Time Frame: 10 years
Rates of cirrhosis, decompensation and hepatocellular carcinoma.
10 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Genotype profile in Chinese patients of each disease
Time Frame: 10 years
10 years
Natural history of Chinese patients with each disease of different genotype
Time Frame: 10 years
10 years
Causes of death in Chinese patients of each disease
Time Frame: 10 years
10 years

Other Outcome Measures

Outcome Measure
Time Frame
Quality of life
Time Frame: 10 years
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Beijing Friendship Hospital

Collaborators

Peking University First Hospital
Fudan University
Beijing YouAn Hospital
Beijing Ditan Hospital
Nanfang Hospital of Southern Medical University
Beijing Anzhen Hospital
Henan Provincial People's Hospital
Hebei Medical University Third Hospital
Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital
Logistics University of Chinese People's Armed Police Forces
West China Second University Hospital of Sichuan University
Jinshan Hospital Fudan University

Investigators

  • Principal Investigator: Jidong Jia, Doctor, Beijing Friendship Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 13, 2015

Primary Completion (Anticipated)

April 28, 2022

Study Completion (Anticipated)

April 28, 2027

Study Registration Dates

First Submitted

April 21, 2017

First Submitted That Met QC Criteria

April 24, 2017

First Posted (Actual)

April 27, 2017

Study Record Updates

Last Update Posted (Actual)

April 27, 2017

Last Update Submitted That Met QC Criteria

April 24, 2017

Last Verified

April 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CR-GMLD

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Genetic/Metabolic Liver Diseases

Clinical Trials on Standard of care

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Turkmenistan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe