A Controlled Study of Potential Therapeutic Effect of Oral Zinc in Manifesting Carriers of Wilson Disease

NCT03659331 | Unknown

• 1 other identifier: 4987-18-SMC
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Wilson Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  9 months

Study Arms (1)

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.

Dietary Supplement: Zinc

Interventions

Zinc DIETARY_SUPPLEMENT

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.

Also known as: blood tests

unexplained elevation of liver enzymes

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• patients over the age of 18 unexplained elevation of liver enzymes patients that carry a single mutation in the ATP7B gene.

Sponsors & Collaborators

• Prof. Elon Pras: lead

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Zinc; Hematologic Tests

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

Intervention Hierarchy (Ancestors)

Metals, Heavy; Elements; Inorganic Chemicals; Transition Elements; Metals; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• Elon Pras, Prof

  The Institute of Human Genetics, Sheba Medical Center, Israel

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Elon Pras, Prof

CONTACT

972-35302998; elon.prasProf@sheba.gov.il

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: head of The Institute of Human Genetics

Model Details: 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

Study Record Dates

• First Submitted: August 28, 2018
• First Posted: September 6, 2018
• Study Start: September 1, 2018
• Primary Completion: August 1, 2019
• Study Completion: October 1, 2019
• Last Updated: September 6, 2018

Record last verified: 2018-09