Wilson's DiseaseNCT04012658
A Registered Cohort Study on Wilson's Disease
An observational study of Wilson's Disease, run by Wan-Jin Chen at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Wilson's Disease
- How many people
- 2,000 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2019to Dec 2049
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients with Wilson's Disease are diagnosed by the clinical and/or genetic features
Who may be able to join
- 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
Who cannot join
- Participants are unable to comply with study procedures and visit schedule
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Fujian Medical University
Fuzhou
Jin He, MD
86-0591-87982772, hejinfjmu@hotmail.com
About this study
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
- Being tested
- No intervention
- Keywords
- Wilson's Disease, Nature history, Genetics
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
No intervention
| Study group | Role | What they receive |
|---|---|---|
| Patients with the genetic diagnosis of Wilson's Disease | Not described Receives: Other: No intervention | |
| Asymptomatic Wilson's Disease carriers | Not described Receives: Other: No intervention | |
| Relatives of Wilson's Disease patients or carriers | Not described Receives: Other: No intervention | |
| Unrelated healthy controls | Not described Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 30years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2019
- Study start1 Jul 2019
- Primary completionDec 2039 (estimated)
- Study completionDec 2049 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wan-Jin Chen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Wan-Jin Chen, First Affiliated Hospital of Fujian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04012658
- Sponsor's protocol ID
- MRCTA,ECFAH OF FMU[2019]197
- Status last verified
- Sep 2019
- First submitted
- 6 Jul 2019
- Last updated
- 23 Sep 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Registered Cohort Study on Wilson's Disease
- ClinicalTrials.gov ID
- NCT04012658
- Acronym / sponsor ID
- MRCTA,ECFAH OF FMU[2019]197
- Conditions
- Wilson's Disease
- Keywords
- Wilson's Disease, Nature history, Genetics
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,000 participants (estimated)
- Lead sponsor
- Wan-Jin ChenUniversity, hospital or other
- Study start
- 1 Jul 2019 (actual)
- Primary completion
- Dec 2039 (estimated)
- Study completion
- Dec 2049 (estimated)
- First posted
- 9 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with Wilson's Disease are diagnosed by the clinical and/or genetic features
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- Participants are unable to comply with study procedures and visit schedule
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionNo intervention | Other | 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 |
Primary outcome
The change of Unified Wilson's Disease Rating Scale (UWDRS)
Time frame: Up to 30years
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Wilson's Disease, run by Wan-Jin Chen at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jin He, MD (86-0591-87982772, hejinfjmu@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of Unified Wilson's Disease Rating Scale (UWDRS) (time frame: Up to 30years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2019.