Back to resultsA Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
Primary Purpose
Wilson Disease
Status
Unknown status
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Zinc
Sponsored by
About this trial
This is an interventional treatment trial for Wilson Disease
Eligibility Criteria
Inclusion Criteria:
patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.
-
Exclusion Criteria:
na
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
unexplained elevation of liver enzymes
Arm Description
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03659331
Brief Title
A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
Official Title
A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
Study Type
Interventional
2. Study Status
Record Verification Date
September 2018
Overall Recruitment Status
Unknown status
Study Start Date
September 2018 (Anticipated)
Primary Completion Date
August 2019 (Anticipated)
Study Completion Date
October 2019 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Prof. Elon Pras
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief 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. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
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)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
unexplained elevation of liver enzymes
Arm Type
Experimental
Arm Description
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.
Intervention Type
Dietary Supplement
Intervention Name(s)
Zinc
Other Intervention Name(s)
blood tests
Intervention Description
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.
Primary Outcome Measure Information:
Title
measurement of liver enzymes in blood tests
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, is statistically significant and supports the association between a single mutation in the ATP7B gene and liver injury.
Time Frame
9 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.
-
Exclusion Criteria:
na
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Elon Pras, Prof
Phone
972-35302998
Email
elon.prasProf@sheba.gov.il
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elon Pras, Prof
Organizational Affiliation
The Institute of Human Genetics, Sheba Medical Center, Israel
Official's Role
Principal Investigator
12. IPD Sharing Statement
Learn more about this trial
A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease
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