NCT03131427

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20,000

participants targeted

Target at P75+ for all trials

Timeline
12mo left

Started Jun 2015

Longer than P75 for all trials

Geographic Reach
1 country

12 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress92%
Jun 2015Apr 2027

Study Start

First participant enrolled

June 13, 2015

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

April 21, 2017

Completed
6 days until next milestone

First Posted

Study publicly available on registry

April 27, 2017

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 28, 2022

Completed
5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 28, 2027

Expected
Last Updated

April 27, 2017

Status Verified

April 1, 2017

Enrollment Period

6.9 years

First QC Date

April 21, 2017

Last Update Submit

April 24, 2017

Conditions

Keywords

Wilson's DiseaseHereditary HemochromatosisHereditary HyperbilirubinemiasInherited Cholestatic Liver DiseaseGenetic Testing

Outcome Measures

Primary Outcomes (1)

  • rate of liver-related events of each disease.

    Rates of cirrhosis, decompensation and hepatocellular carcinoma.

    10 years

Secondary Outcomes (3)

  • 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

Other Outcomes (1)

  • Quality of life

    10 years

Study Arms (5)

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

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

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

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

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

Interventions

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

Hereditary HemochromatosisHereditary HyperbilirubinemiasInherited Cholestatic Liver DiseaseOther genetic/metabolic liver diseasesWilson's Disease

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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.

You may not qualify if:

  • Patients who are unable or unwilling to provide informed consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Beijing Ditan Hospital

Beijing, Beijing Municipality, 100015, China

RECRUITING

Beijing Anzhen Hospital, Capital Medical University

Beijing, Beijing Municipality, 100029, China

RECRUITING

Peking University First Hospital

Beijing, Beijing Municipality, 100034, China

RECRUITING

Beijing YouAn Hospital

Beijing, Beijing Municipality, 100069, China

RECRUITING

Nanfang Hospital of Southern Medical University

Guangzhou, Guangdong, 510515, China

RECRUITING

Hebei Medical University Third Hospital

Shijiazhuang, Hebei, 050051, China

RECRUITING

Henan Provincial Hospital

Zhengzhou, Henan, 450003, China

RECRUITING

Zhongshan Hospital, Fudan University

Shanghai, Shanghai Municipality, 200032, China

RECRUITING

Jinshan Hospital of Fudan University

Shanghai, Shanghai Municipality, 201508, China

RECRUITING

West China Women's and Children's Hospital, Sichuan University

Chengdu, Sichuan, 610041, China

RECRUITING

Affiliated Hospital, Logistics University of People's Armed Police Force

Tianjin, Tianjin Municipality, 300163, China

RECRUITING

Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital

Ürümqi, Xinjiang, 830099, China

RECRUITING

Related Publications (3)

  • Xu A, Lv T, Zhang B, Zhang W, Ou X, Huang J. Development and evaluation of an unlabeled probe high-resolution melting assay for detection of ATP7B mutations in Wilson's disease. J Clin Lab Anal. 2017 Jul;31(4):e22064. doi: 10.1002/jcla.22064. Epub 2016 Sep 17.

    PMID: 27638368BACKGROUND
  • Lv T, Li X, Zhang W, Zhao X, Ou X, Huang J. Recent advance in the molecular genetics of Wilson disease and hereditary hemochromatosis. Eur J Med Genet. 2016 Oct;59(10):532-9. doi: 10.1016/j.ejmg.2016.08.011. Epub 2016 Aug 31.

    PMID: 27592149BACKGROUND
  • Zhang W, Li Y, Xu A, Ouyang Q, Wu L, Zhou D, Wu L, Zhang B, Zhao X, Wang Y, Wang X, Duan W, Wang Q, You H, Huang J, Ou X, Jia J; China Registry of Genetic/Metabolic Liver Diseases (CR-GMLD) Group. Identification of novel non-HFE mutations in Chinese patients with hereditary hemochromatosis. Orphanet J Rare Dis. 2022 Jun 6;17(1):216. doi: 10.1186/s13023-022-02349-y.

Biospecimen

Retention: SAMPLES WITH DNA

whole blood, serum

MeSH Terms

Conditions

Hepatolenticular DegenerationHemochromatosisHyperbilirubinemia, Hereditary

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetal Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesIron OverloadIron Metabolism Disorders

Intervention Hierarchy (Ancestors)

Quality Indicators, Health CareQuality of Health CareHealth Services AdministrationHealth Care Quality, Access, and Evaluation

Study Officials

  • Jidong Jia, Doctor

    Beijing Friendship Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Jidong Jia, MD

CONTACT

Xiaojuan Ou, MD

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
10 Years
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Liver Research Center

Study Record Dates

First Submitted

April 21, 2017

First Posted

April 27, 2017

Study Start

June 13, 2015

Primary Completion

April 28, 2022

Study Completion (Estimated)

April 28, 2027

Last Updated

April 27, 2017

Record last verified: 2017-04

Data Sharing

IPD Sharing
Will not share

Locations