Wilson DiseaseNCT03659331
A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
A study testing Zinc in people with Wilson Disease, run by Prof. Elon Pras.
Trial summary
The assumption is that in some of the carriers, the increase in enzymes reflects tissue damage due to excess copper. The reduction of the amount of copper absorbed will decrease excess copper in the liver, which will result in a decrease in the level of liver enzymes. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Wilson Disease
- What is being tested?
- Zinc
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- No sites listed yet
- Study dates
- Sep 2018to Oct 2019
- Phase
- Interventional
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.
- -
Who cannot join
- na
Trial locations and contacts
No sites are listed yet.
About this study
The assumption is that in some of the carriers, the increase in enzymes reflects tissue damage due to excess copper. The reduction of the amount of copper absorbed will decrease excess copper in the liver, which will result in a decrease in the level of liver enzymes. Zinc causes the induction of metalothionines in the intestine, which in turn prevents absorption of copper from the digestive system. Zinc administration in Wilson's patients causes the depletion of copper deposits and constitutes one of the cornerstones in the treatment of this disease.
Read the detailed description
The research group is composed of patients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again.
- Conditions
- Wilson Disease
- Being tested
- Zinc
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZincDietary supplement
Also called blood tests
blood tests will be performed: GGT SGOT, SGPT, LDH, A.P. Direct and indirect bilirubin, blood proteins, cholesterol, P.T. and a complete blood count.
| Study group | Role | What they receive |
|---|---|---|
| unexplained elevation of liver enzymes | Experimental | patients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again. Receives: Dietary Supplement: Zinc |
What is being measured
The primary outcome is the main question the study is designed to answer.
measurement of liver enzymes in blood tests
A model based on the assumption that at least 50 subjects will be recruited, and assuming that 50% of the patients will have a significant reduction of liver enzymes, is statistically significant and supports the association between a single mutation in the ATP7B gene and liver injury.
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2018
- Study startSep 2018 (estimated)
- Primary completionAug 2019 (estimated)
- Study completionOct 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A model based on the assumption that at least 50 subjects will be recruited, and assuming that 50% of the patients will have a significant reduction of liver enzymes
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Prof. Elon Pras
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Prof. Elon Pras, Sheba Medical Center
- Investigators
- Elon Pras, Prof, Principal investigator, The Institute of Human Genetics, Sheba Medical Center, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03659331
- Sponsor's protocol ID
- 4987-18-SMC
- Status last verified
- Sep 2018
- First submitted
- 28 Aug 2018
- Last updated
- 6 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
- ClinicalTrials.gov ID
- NCT03659331
- Acronym / sponsor ID
- 4987-18-SMC
- Conditions
- Wilson Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Prof. Elon PrasOther government
- Study start
- Sep 2018 (estimated)
- Primary completion
- Aug 2019 (estimated)
- Study completion
- Oct 2019 (estimated)
- First posted
- 6 Sep 2018 (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 assumption is that in some of the carriers, the increase in enzymes reflects tissue damage due to excess copper. The reduction of the amount of copper absorbed will decrease excess copper in the liver, which will result in a decrease in the level of liver enzymes. Zinc causes the induction of metalothionines in the intestine, which in turn prevents absorption of copper from the digestive system. Zinc administration in Wilson's patients causes the depletion of copper deposits and constitutes one of the cornerstones in the treatment of this disease.
Detailed description
The research group is composed of patients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A model based on the assumption that at least 50 subjects will be recruited, and assuming that 50% of the patients will have a significant reduction of liver enzymes
| Arm | Type | Receives |
|---|---|---|
| unexplained elevation of liver enzymes | Experimental | Dietary Supplement: Zincpatients over the age of 18 referred for unexplained elevation of liver enzymes and carry a single mutation in the ATP7B gene. After a washout period of 3 months these patients will be re-checked for liver enzymes and if high will receive zinc therapy at a dose of 300 mg / day for 6 months, after which the liver enzymes will be checked again. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.
- -
Key exclusion criteria (1 listed)
- na
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 |
|---|---|---|
| ZincAlso: blood testsblood tests will be performed: GGT SGOT, SGPT, LDH, A.P. Direct and indirect bilirubin, blood proteins, cholesterol, P.T. and a complete blood count. | Dietary supplement | unexplained elevation of liver enzymes |
Primary outcome
measurement of liver enzymes in blood tests
Time frame: 9 months
A model based on the assumption that at least 50 subjects will be recruited, and assuming that 50% of the patients will have a significant reduction of liver enzymes, is statistically significant and supports the association between a single mutation in the ATP7B gene and liver injury.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2018.
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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.
Elon Pras, Prof principal investigator
The Institute of Human Genetics, Sheba Medical Center, Israel
Questions about this trial
What is this clinical trial studying?
A study testing Zinc in people with Wilson Disease, run by Prof. Elon Pras.
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 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: status not confirmed, as of the sponsor's last update on 6 Sep 2018. 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?
Contact Elon Pras, Prof (972-35302998, elon.prasProf@sheba.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
measurement of liver enzymes in blood tests (time frame: 9 months). 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 6 Sep 2018.