Research on the Clinical Characteristics and Key Diagnosis and Treatment Technology of Genetic and Metabolic Liver Disease

NCT ID: NCT05601557

Last Updated: 2022-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

480 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-12-01

Study Completion Date

2025-04-30

Brief Summary

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1. Establish a follow-up cohort of genetic and metabolic liver disease in The Chinese population, and carry out research on disease spectrum, clinical characteristics and personalized diagnosis and treatment to improve the level of diagnosis and treatment.
2. Establish a multidisciplinary collaborative diagnosis and treatment model of genetic metabolic liver disease, develop and promote diagnosis and treatment paths, and improve the diagnosis and treatment ability of genetic metabolic liver disease in Beijing and even the whole country.
3. Establish a new CRISPR gene diagnosis technology to realize fast and low-cost genetic testing.
4. Elucidating the genetic mutation spectrum of common genetic and metabolic liver disease in China is helpful to accurate gene diagnosis and functional research.
5. Study the genotype-phenotype, mutation and clinical outcome relationship and influencing factors of the common genetic and metabolic liver disease population in China, to guide the early diagnosis, early treatment and improve the prognosis.

Detailed Description

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Conditions

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Diagnosis , Treatment, Genetic and Metabolic Liver Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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hyperbilirubinemia

Gilbert syndrome Crigler-Najjar syndrome Dubin-Johnson syndrome Rotor syndrome PFIC BIRC

There is no intervention

Intervention Type OTHER

The genotype of the patients was analyzed.

Wilson disease

Leipzig score system was used for diagnosis, and the total score ≥4 points could be confirmed. The total score of 3 is suspected diagnosis, which requires further examination. A total score of 2 or less is not considered for diagnosis.

There is no intervention

Intervention Type OTHER

The genotype of the patients was analyzed.

Hemochromatosis

① clinical manifestations of extensive skin pigmentation, bronzing; Decline to disappearance of sexual function; Mild hepatosplenomegaly, may appear jaundice; The heart is enlarged; Pain and swelling mainly in metacarpophalangeal joints; Decreased glucose tolerance and increased blood glucose; ② Serum iron was significantly increased, serum transferrin was normal or decreased, transferrin saturation was significantly increased, often more than 62%, serum ferritin was significantly increased, often more than 500ug/L; (3)/HJV/HAMP TFR2 / SLC40A1 HFE gene mutation.

There is no intervention

Intervention Type OTHER

The genotype of the patients was analyzed.

Glycogen accumulation disease

According to different types, there may be the following manifestations, which need specific analysis. ① Clinical manifestations of abdominal distension, fasting hypoglycemia and other symptoms; ② Laboratory examination showed metabolic acidosis, hyperlactic acidemia, hyperuricemia and hyperlipidemia; ③ Abdominal CT showed enlarged liver volume; ④ Serum glucosidase activity decreased; (5) the GAA/G6PC/SLC374A/AGL/PYG/PHK gene mutations.

There is no intervention

Intervention Type OTHER

The genotype of the patients was analyzed.

Other types of inherited metabolic liver disease

There is no intervention

Intervention Type OTHER

The genotype of the patients was analyzed.

Interventions

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There is no intervention

The genotype of the patients was analyzed.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

Meet the diagnostic criteria of each disease.

Exclusion Criteria

1. Co-infected with hepatitis B virus, hepatitis C virus and HIV;
2. Patients with liver fibrosis and cirrhosis caused by other causes;
3. Patients with alcoholic liver disease and autoimmune liver disease;
4. Liver malignancy has been suggested or confirmed by evidence;
5. Combined with other serious systemic diseases.
Minimum Eligible Age

0 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Sujun Zheng

OTHER

Sponsor Role lead

Responsible Party

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Sujun Zheng

Ethics Committee Member

Responsibility Role SPONSOR_INVESTIGATOR

Other Identifiers

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LL-2022-048-K

Identifier Type: -

Identifier Source: org_study_id

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