Clinical Outcome in WILSON DiseaseNCT05792319
Clinical Profile and Out Come of Children With Wilson's Disease
An observational study of Clinical Outcome in WILSON Disease, run by Assiut University.
Trial summary
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Clinical Outcome in WILSON Disease
- What is being tested?
- 24 hour copper in urine and liver function test
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 1 Mar 2024to 30 Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: The aim of this study to describe the clinical profile of children diagnosed as Wilson's disease who were admitted to A.U.C.H and explore the out come of these patients either they receive the chelation therapy or not along a period of one year
Who may be able to join
- All patients aged 1-18 years
- fulfill the criteria for diagnosis of Wilson's disease according to Wilson's score
Who cannot join
- persistent neonatal jaundice.
- Patients presented with Acute and chronic liver disease rather than Wilson's e.g
- Autoimmune hepatitis
- Hepatitis C virus ,
- Hepatitis B virus infection ,(d)Hepatic focal lesions and(e) Metabolic liver disease e.g Gauchier ,glycogen storage disease and Niemman pick .
About this study
Official title: Clinical Profile and Out Come of Children With Wilson's Disease :A Single Tertiary Center Study .
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study
Read the detailed description
Wilson disease (WD) is an autosomal-recessive disorder of copper metabolism caused by mutations in the ATP7B gene . It presents in childhood, adolescence or adulthood with a wide range of clinical manifestations. The disease prevalence has previously been estimated as 1 in 30,000 , but some recent analyses have suggested a genetic prevalence of 1 in 7,000 . Copper is absorbed from the stomach and duodenum, taken up by the liver, and secreted by the liver into the systemic circulation bound to ceruloplasmin ATP7B transports copper through the trans-Golgi network in hepatocytes before it is incorporated into apoceruloplasmin which is secreted as holoceruloplasmin.
ATP7B is also essential for biliary excretion of copper when cytoplasmic levels are high. Dysfunction of ATP7B therefore leads to accumulation of copper in the liver giving rise to cellular damage and disease, and the release of nonceruloplasmin bound copper into the systemic circulation. Copper also accumulates and is associated with cellular damage and disease in other organs mostly in the brain Typical presentation of WD is in adolescence to early adulthood, but it may occur at any age . Clinical presentation can vary widely in terms of type and severity, but the key features are various degrees of liver disease,
- Conditions
- Clinical Outcome in WILSON Disease
- Being tested
- 24 hour copper in urine and liver function test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
24 hour copper in urine and liver function testDiagnostic test
sampling 24 hour copper in urine and liver function test
What is being measured
The primary outcome is the main question the study is designed to answer.
COC in WILSON disease
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2023
- Study start1 Mar 2024 (estimated)
- Primary completion30 Oct 2024 (estimated)
- Study completion30 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Clinical Outcome in WILSON DiseaseStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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
- Case-control
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Rehab Ahmed Ali, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05792319
- Sponsor's protocol ID
- COC of WILSON disease
- Status last verified
- Nov 2023
- First submitted
- 9 Feb 2023
- Last updated
- 28 Feb 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Tryambak S, Sumanta L, Radheshyam P, Sutapa G. Clinical profile, prognostic indicators and outcome of Wilson's disease in children: a hospital based study. Trop Gastroenterol. 2009 Jul-Sep;30(3):163-6.
PubMed 20306753
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Profile and Out Come of Children With Wilson's Disease :A Single Tertiary Center Study .
- ClinicalTrials.gov ID
- NCT05792319
- Acronym / sponsor ID
- COC of WILSON disease
- Conditions
- Clinical Outcome in WILSON Disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Mar 2024 (estimated)
- Primary completion
- 30 Oct 2024 (estimated)
- Study completion
- 30 Oct 2024 (estimated)
- First posted
- 31 Mar 2023 (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.
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study
Detailed description
Wilson disease (WD) is an autosomal-recessive disorder of copper metabolism caused by mutations in the ATP7B gene . It presents in childhood, adolescence or adulthood with a wide range of clinical manifestations. The disease prevalence has previously been estimated as 1 in 30,000 , but some recent analyses have suggested a genetic prevalence of 1 in 7,000 . Copper is absorbed from the stomach and duodenum, taken up by the liver, and secreted by the liver into the systemic circulation bound to ceruloplasmin ATP7B transports copper through the trans-Golgi network in hepatocytes before it is incorporated into apoceruloplasmin which is secreted as holoceruloplasmin.
ATP7B is also essential for biliary excretion of copper when cytoplasmic levels are high. Dysfunction of ATP7B therefore leads to accumulation of copper in the liver giving rise to cellular damage and disease, and the release of nonceruloplasmin bound copper into the systemic circulation. Copper also accumulates and is associated with cellular damage and disease in other organs mostly in the brain Typical presentation of WD is in adolescence to early adulthood, but it may occur at any age . Clinical presentation can vary widely in terms of type and severity, but the key features are various degrees of liver disease,
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The aim of this study to describe the clinical profile of children diagnosed as Wilson's disease who were admitted to A.U.C.H and explore the out come of these patients either they receive the chelation therapy or not along a period of one year
Key inclusion criteria (2 listed)
- All patients aged 1-18 years
- fulfill the criteria for diagnosis of Wilson's disease according to Wilson's score
Key exclusion criteria (5 listed)
- persistent neonatal jaundice.
- Patients presented with Acute and chronic liver disease rather than Wilson's e.g
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 |
|---|---|---|
| 24 hour copper in urine and liver function testsampling 24 hour copper in urine and liver function test | Diagnostic test | – |
Primary outcome
COC in WILSON disease
Time frame: Baseline
Clinical profile and out come of Children with Wilson's disease :A single tertiary center study
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2024.
- Background: PubMed 20306753Tryambak S, Sumanta L, Radheshyam P, Sutapa G. Clinical profile, prognostic indicators and outcome of Wilson's disease in children: a hospital based study. Trop Gastroenterol. 2009 Jul-Sep;30(3):163-6.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Clinical Outcome in WILSON Disease, run by Assiut University.
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 2 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 28 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
COC in WILSON disease (time frame: Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05792319, which also lists 1 linked publication.
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 28 Feb 2024.