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China Registry for Genetic / Metabolic Liver Diseases

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ClinicalTrials.gov Identifier: NCT03131427
Recruitment Status : Recruiting
First Posted : April 27, 2017
Last Update Posted : April 27, 2017
Sponsor:
Collaborators:
Beijing YouAn Hospital
Henan Provincial 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
Information provided by (Responsible Party):
Jidong Jia, Beijing Friendship Hospital

Tracking Information
First Submitted Date April 21, 2017
First Posted Date April 27, 2017
Last Update Posted Date April 27, 2017
Actual Study Start Date June 13, 2015
Estimated Primary Completion Date April 28, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 24, 2017)
rate of liver-related events of each disease. [ Time Frame: 10 years ]
Rates of cirrhosis, decompensation and hepatocellular carcinoma.
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: April 24, 2017)
  • Genotype profile in Chinese patients of each disease [ Time Frame: 10 years ]
  • Natural history of Chinese patients with each disease of different genotype [ Time Frame: 10 years ]
  • Causes of death in Chinese patients of each disease [ Time Frame: 10 years ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures
 (submitted: April 24, 2017)
Quality of life [ Time Frame: 10 years ]
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title China Registry for Genetic / Metabolic Liver Diseases
Official Title A Nation-wide Hospital-based Registry:China Registry for Genetic / Metabolic Liver Diseases
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.
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 10 Years
Biospecimen Retention:   Samples With DNA
Description:
whole blood, serum
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.
Condition Genetic/Metabolic Liver Diseases
Intervention Drug: Standard of care
Standard of care according to the updated national and/or international guidelines
Study Groups/Cohorts
  • 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.
    Intervention: Drug: Standard of care
  • 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.
    Intervention: Drug: Standard of care
  • 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).
    Intervention: Drug: Standard of care
  • 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).
    Intervention: Drug: Standard of care
  • 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.
    Intervention: Drug: Standard of care
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 24, 2017)
20000
Original Estimated Enrollment Same as current
Estimated Study Completion Date April 28, 2027
Estimated Primary Completion Date April 28, 2022   (Final data collection date for primary outcome measure)
Eligibility 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.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Jidong Jia, MD 010-63139816 jia_jd@ccmu.edu.cn
Contact: Xiaojuan Ou, MD 010-63138315 ouxj16@sina.com
Listed Location Countries China
Removed Location Countries  
 
Administrative Information
NCT Number NCT03131427
Other Study ID Numbers CR-GMLD
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Jidong Jia, Beijing Friendship Hospital
Study Sponsor Beijing Friendship Hospital
Collaborators
  • Beijing YouAn Hospital
  • Henan Provincial 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
Investigators
Principal Investigator: Jidong Jia, Doctor Beijing Friendship Hospital
PRS Account Beijing Friendship Hospital
Verification Date April 2017