A Registered Cohort Study on Wilson's Disease

NCT04012658 | Recruiting DMC

• 1 other identifier: MRCTA,ECFAH OF FMU[2019]197
• Study Type: observational
• Target: 2,000
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to determine the clinical spectrum and natural progression of Wilson's Disease in a prospective multicenter natural history study, to assess the clinical, genetic, epigenetic features and biomarkers of patients with Wilson's Disease to optimize clinical management.

Conditions

Wilson's Disease

Keywords

Wilson's Disease; Nature history; Genetics

Outcome Measures

Primary Outcomes (1)

• The change of Unified Wilson's Disease Rating Scale (UWDRS)

  Disease severity will be assessed by application of the Unified Wilson's Disease Rating Scale (UWDRS), a clinical rating scale consists of three subscales. Higher UWDRS total scores indicate more severe disease.

  Up to 30years

Study Arms (4)

Patients with the genetic diagnosis of Wilson's Disease

Other: No intervention

Asymptomatic Wilson's Disease carriers

Other: No intervention

Relatives of Wilson's Disease patients or carriers

Other: No intervention

Unrelated healthy controls

Other: No intervention

Interventions

No intervention OTHER

No intervention

Asymptomatic Wilson's Disease carriers; Patients with the genetic diagnosis of Wilson's Disease; Relatives of Wilson's Disease patients or carriers; Unrelated healthy controls

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Wilson's Disease are diagnosed by the clinical and/or genetic features

You may qualify if:

• Patients with the genetic diagnosis of Wilson's Disease
• Asymptomatic Wilson's Disease carriers
• Relatives of Wilson's Disease patients or carriers
• Unrelated healthy controls
• Participants or Parent(s)/legal guardian(s) willing and able to complete the informed consent process

You may not qualify if:

• \* Participants are unable to comply with study procedures and visit schedule

Sponsors & Collaborators

• Wan-Jin Chen: lead

Study Sites (1)

First Affiliated Hospital of Fujian Medical University

Fuzhou, China

RECRUITING

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Jin He, MD

CONTACT

86-0591-87982772; hejinfjmu@hotmail.com

Yi Lin, MD.PhD

CONTACT

8613615039153; linyi7811@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: The Vice-Director for the Department of Neurology

Study Record Dates

• First Submitted: July 6, 2019
• First Posted: July 9, 2019
• Study Start: July 1, 2019
• Primary Completion (Estimated): December 1, 2039
• Study Completion (Estimated): December 1, 2049
• Last Updated: September 23, 2019

Record last verified: 2019-09

Locations

CN (1)