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

China Registry for Genetic / Metabolic Liver Diseases

Sponsors

Lead sponsor: Beijing Friendship Hospital

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

Source Beijing Friendship Hospital
Brief Summary

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.

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.

Overall Status Recruiting
Start Date June 13, 2015
Completion Date April 28, 2027
Primary Completion Date April 28, 2022
Study Type Observational [Patient Registry]
Primary Outcome
Measure Time Frame
rate of liver-related events of each disease. 10 years
Secondary Outcome
Measure Time Frame
Genotype profile in Chinese patients of each disease 10 years
Natural history of Chinese patients with each disease of different genotype 10 years
Causes of death in Chinese patients of each disease 10 years
Enrollment 20000
Condition
Intervention

Intervention type: Drug

Intervention name: Standard of care

Description: Standard of care according to the updated national and/or international guidelines

Eligibility

Sampling method: Non-Probability Sample

Criteria:

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.

Gender: All

Minimum age: N/A

Maximum age: N/A

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Jidong Jia, Doctor Principal Investigator Beijing Friendship Hospital
Overall Contact

Last name: Jidong Jia, MD

Phone: 010-63139816

Email: [email protected]

Location
facility status contact
Beijing Ditan Hospital | Beijing, Beijing, 100015, China Recruiting Wen Xie, MD [email protected]
Beijing Anzhen Hospital, Capital Medical University | Beijing, Beijing, 100029, China Recruiting Yanwen Qin, MD [email protected]
Peking University First Hospital | Beijing, Beijing, 100034, China Recruiting Yanling Yang, MD [email protected]
Beijing YouAn Hospital | Beijing, Beijing, 100069, China Recruiting Sujun Zheng, MD [email protected]
Nanfang Hospital of Southern Medical University | Guangzhou, Guangdong, 510515, China Recruiting Yongpeng Chen, MD [email protected]
Hebei Medical University Third Hospital | Shijiazhuang, Hebei, 050051, China Recruiting Yuemin Nan, MD [email protected]
Henan Provincial Hospital | Zhengzhou, Henan, 450003, China Recruiting Jia Shang, MD [email protected]
Zhongshan Hospital, Fudan University | Shanghai, Shanghai, 200032, China Recruiting Wei Jiang, MD [email protected]
Jinshan Hospital of Fudan University | Shanghai, Shanghai, 201508, China Recruiting Jianshe Wang, MD [email protected]
West China Women's and Children's Hospital, Sichuan University | Chengdu, Sichuan, 610041, China Recruiting Chaomin Wan, MD [email protected]
Affiliated Hospital, Logistics University of People's Armed Police Force | Tianjin, Tianjin, 300163, China Recruiting Hai Li, MD [email protected]
Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital | Urumqi, Xinjiang, 830099, China Recruiting Xiaozhong Wang, MD [email protected]
Location Countries

China

Verification Date

April 2017

Responsible Party

Responsible party type: Principal Investigator

Investigator affiliation: Beijing Friendship Hospital

Investigator full name: Jidong Jia

Investigator title: Liver Research Center

Keywords
Has Expanded Access No
Condition Browse
Arm Group

Arm group label: Wilson's Disease

Description: 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.

Arm group label: Hereditary Hemochromatosis

Description: 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.

Arm group label: Hereditary Hyperbilirubinemias

Description: 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).

Arm group label: Inherited Cholestatic Liver Disease

Description: Patients who were diagnosed or possibly diagnosed with Inherited cholestatic liver disease, including progressive familial intrahepatic cholestasis(PFIC) and benign recurrent intrahepatic cholestasis(BRIC).

Arm group label: Other genetic/metabolic liver diseases

Description: 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.

Patient Data No
Study Design Info

Observational model: Cohort

Time perspective: Prospective

Source: ClinicalTrials.gov