Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C

November 18, 2014 updated by: Ari Zimran, Shaare Zedek Medical Center

Enhanced HCV Nonstructural Protein 3 (NS3) -Specific T Cell Proliferation,Interferon γ (IFNγ) and Interleukin-10 (IL-10) Secreting Clones, and Peripheral Blood Natural Killers T Cells ( NKT Cells) in Patients With Type I Gaucher Disease Infected With HCV : An Advantage in Anti Hepatitis Immunity?

Study objectives:

  • Investigate the anti-HCV response in patients with Gaucher disease(GD)
  • Define the potential role of high levels of Glucocerebroside in the immune system

Study hypothesis:

High levels of Glucocerebroside can be used as a tool in the antiviral treatment of hepatitis C by potentiating the immune response of natural killer T cells and dendritic cells

Study Overview

Status

Unknown

Conditions

Detailed Description

Gaucher disease is the most common glycolipid storage disorder, caused by reduced activity of the lysosomal enzyme glucocerebrosidase, which leads to the accumulation of the substrate, glucocerebroside (GC), in the cells of the reticulo-endothelial system.

One of the hallmarks of GD is its great phenotypic heterogeneity with variable presentations and symptoms, beginning with a lethal variant of infants dying at or near birth with hydrops fetalis and ichthyoids at one extreme and totally asymptomatic individuals without any physical or laboratory abnormalities at the other extreme.

This autosomal recessive disease is pan-ethnic, but it is especially prevalent among Ashkenazi Jews. From over 300 different mutations reported in the glucocerebrosidase gene, five account for 98% of the disease-producing alleles. Of these mutations, N370S (or 1226G) occurs in 1 out of 17 Ashkenazi individuals, leading to a disease frequency of 1:850 in this ethnic group.

The high prevalence of more than a single mutation among Ashkenazi Jews and the existence of two additional rare inherited lysosomal glycolipid storage diseases, Tay Sachs and Nieman Pick, at a higher prevalence within the same ethnic group is believed to be caused by selective advantage.

Available genetic data are consistent with a founder effect(4) whereas the nature of such an advantage has not been identified.

The aim of this study was to investigate the anti-HCV immune response in patients with GD in an attempt to define the potential role of high levels of GC in the immune system and antiviral immunity.

Study importance:

The host metabolic background exerts a profound effect on antiviral immunity, which may influence the clinical course of chronic HCV infection.

The accumulation of GC in patients with GD may provide a selective evolutionary advantage to these patients.

Glucocerebroside was recently tested in human trials and shown to be effective in altering NKT- dependent metabolic pathways, insulin resistance, and associated liver injury.

The present study examine the capability of Glucocerebroside to be be used as a tool in the antiviral treatment of hepatitis C by potentiating the immune response of natural killer T cells and dendritic cells.

Statistical Analysis:

Data are presented as the mean ± standard deviation (SD). The Kruskal Wallis non-parametric ANOVA test was used to identify differences between the study groups.

The student t-test and non-parametric Mann-Whitney test were used to compare quantitative variables between the study groups as appropriate; P <0.05 was considered to be significant.

Study Type

Observational

Enrollment (Anticipated)

80

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Israel
      • Jerusalem, Israel
        • Recruiting
        • Hadassah Medical Center
        • Contact:
        • Sub-Investigator:
          • Yaron Ilan, Prof.
      • Jerusalem, Israel, 91120
        • Recruiting
        • Shaare Zedek , Medical Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Bernardo Melamud, Dr.
        • Sub-Investigator:
          • Ari Zimran, Prof.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 90 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Patients with Gaucher Disease were recruited from the Sha'are Zedek Gaucher disease clinic, a national referral center for the disease.

Description

Inclusion Criteria:

  • Patients with Gaucher disease
  • Patients with hepatitis C without Gaucher disease
  • Individuals or patients without Gaucher disease and hepatitis C
  • Individuals or patients who signed an approval for the research
  • Men and women 18< years of age , pregnant women

Exclusion Criteria:

  • Inabillity to give an approval for the research
  • Acute liver disease that can alter the lab results , such as: Rt. congestive heart failure

severe infection ,inflammation, medication such as : Statins , Isoniazid ,

Amiodarone

- Patients who received treatment for hepatitis C such as: Interferon ,

Pegylated interferon , Ribavirin

-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Gaucher Disease with Hepatitis C

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Gaucher patients' immune system provide enhanced immunity against hepatitis c virus
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Time Frame
the role Glucocerebroside level have by enhanced immunity in patients with Gaucher disease
Time Frame: 30 days
30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shaare Zedek Medical Center

Investigators

  • Principal Investigator: Bernardo Melamud, Dr., Gaucher Clinic , Shaare zedek Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2011

Primary Completion (Anticipated)

March 1, 2016

Study Completion (Anticipated)

April 1, 2016

Study Registration Dates

First Submitted

January 10, 2011

First Submitted That Met QC Criteria

January 10, 2011

First Posted (Estimate)

January 11, 2011

Study Record Updates

Last Update Posted (Estimate)

November 19, 2014

Last Update Submitted That Met QC Criteria

November 18, 2014

Last Verified

November 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SZMC- 89/10

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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